Neurology & Neurosurgery

Mapping the brain’s memory gradient: How the hippocampus helps children form lasting memories

illustration of the hippocampus

As children mature into adolescence, their hippocampus becomes increasingly specialized along its longitudinal axis, contributing to the development of memory and other cognitive functions.

In a study published in PNAS (Proceedings of the National Academy of Sciences), researchers found that the hippocampal gradient and its distinct connections with cortical systems are essential for memory development in children.

“This study is important because it helps us understand how the hippocampus develops and how it communicates with the rest of the brain to form episodic memory, or long-term memories of specific events,” said Hua (Oliver) Xie, PhD, research faculty at Children’s National Hospital and assistant professor at The George Washington University.

The big picture

Researchers looked at two resting-state functional magnetic resonance imaging (fMRI) datasets. The primary dataset included 598 participants between the ages of 8 and 21 and a secondary replication dataset of 196 participants from the Nathan Kline Institute (NKI)-Rockland Sample. Participants were scanned for a total of 26 minutes in resting-state where they stayed still, awake and looked at the fixation crosshair.

The results showed that the head and tail of the hippocampus form distinct communication patterns with other brain regions, which can help predict how well children form episodic memories.

As children mature into adolescence, their hippocampus becomes increasingly specialized along its longitudinal axis, contributing to the development of memory and other cognitive functions.

The hold up in the field

Previous studies have primarily focused on the hippocampus, with many researchers treating the anterior and posterior regions as distinct areas of study. By exploring how the hippocampus communicates with other parts of the brain, this study reveals a more comprehensive understanding than before.

“Our research shows that in children, it’s more of a gradual transition of function along the hippocampus’s long axis, like a color gradient rather than two distinct blocks,” Dr. Xie said.

You can read the full study, Longitudinal hippocampal axis in large-scale cortical systems underlying development and episodic memory, in PNAS.

Additional authors from Children’s National include Venkata Sita Priyanka Illapani, MS, Lauren T. Reppert, BS, Xiaozhen You, PhD, Manu Krishnamurthy, MS, Madison M. Berl, PhD, William D. Gaillard, MD, Leigh N. Sepeta, PhD

Children’s National Hospital designated Pediatric Center of Excellence in Rare Neuroimmune Disorders

Pediatric Center of Excellence in Rare Neuroimmune Disorders seal

Children’s National Hospital’s Neuroimmunology Program has been named a Pediatric Center of Excellence in Rare Neuroimmune Disorders by the Siegel Rare Neuroimmune Association.

The Neuroimmunology Program at Children’s National Hospital has been recognized by the Siegel Rare Neuroimmune Association (SRNA) as a Pediatric Center of Excellence in Rare Neuroimmune Disorders (CERND). This designation highlights the program’s expertise in caring for children with complex neuroimmune conditions and its commitment to advancing research and education in the field.

The program is led by Elizabeth Wells, MD, Senior vice president of the Neuroscience and Behavioral Medicine Center, Alexandra Kornbluh, MD, director of Neuroimmunology Investigational Therapeutics, and Ilana Kahn, MD, clinical director of Neuroimmunology. Together, they oversee a multidisciplinary team providing state-of-the-art diagnosis, treatment and long-term management for children with antibody-mediated autoimmune encephalitis (AE), acute disseminated encephalomyelitis (ADEM), febrile infection related epilepsy syndrome (FIRES), multiple sclerosis (MS), neuromyelitis optica spectrum disorders (NMOSD), myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD), opsoclonus myoclonus ataxia syndrome (OMAS), transverse myelitis (TM), optic neuritis (ON), primary CNS vasculitis and other central nervous system demyelinating and inflammatory conditions.

As a designated CERND, Children’s National offers:

  • Expert-led care: Specialists at the forefront of clinical practice and research.
  • Comprehensive services: Coordinated care across neurology, neurosurgery, neuropsychology, psychiatry, psychology, neuroradiology, physical medicine and rehabilitation, critical care, social work, pharmacy and speech therapy.
  • Research opportunities: Access to clinical trials and studies that drive progress in therapies and patient outcomes.

In addition to delivering high-quality clinical care, the program partners with SRNA to expand educational resources for patients, families and clinicians. Its work aligns with SRNA’s broader objectives to strengthen the CERND network, support innovative research and improve both acute therapies and long-term management strategies for rare neuroimmune disorders.

Advancing innovative treatments for neurofibromatosis type 1

Gilbert Family Foundation logoA new $8 million investment from the Gilbert Family Foundation will support groundbreaking research on neurofibromatosis type 1 (NF1) at Children’s National Hospital. The generous five-year grant continues the Foundation’s longstanding research partnership with the hospital. It will accelerate investigations aimed at improving the lives of children and young adults affected by this genetic condition.

“This grant will enable us to expand basic science, translational and clinical research. It will help us perform two to three innovative clinical trials in the next five years — trials we will share with the NF community as a whole,” says Roger J. Packer, MD, the Gilbert Family Distinguished Professor of Neurofibromatosis at Children’s National.

Dr. Packer will lead the research team, which also includes Javad Nazarian, PhD, Steven Stasheff, MD, Karin S. Walsh, PsyD and Sridevi Yadavilli, MD, PhD.

Why this matters

NF1 is one of the most common genetic conditions in young people. It affects approximately 1 in 2,500 children born each year. It causes potentially deadly nervous system tumors and can pose lifelong health complications. These include vision and learning disabilities, skeletal abnormalities and increased cancer risks.

Dive deeper

The new funding will:

  • Fuel clinical trials testing new therapies that combine MEK inhibitors, which block tumor growth, and checkpoint inhibitors, which help the immune system attack cancer, to treat aggressive NF1 brain tumors. Trials will target high-grade astrocytomas with piloid features (HGAPs) and recalcitrant gliomas. The goal is to improve outcomes for these aggressive tumors by enhancing the body’s immune response to cancer.
  • Drive cognitive research to understand learning, behavior and social deficits in children with NF1.
  • Better detect and understand vision loss in children with optic pathway tumors. Discoveries will help doctors predict, treat and prevent disease progression as well as assess the potential benefits of treatment.
  • Build novel lab models to supercharge drug discovery. The grant will support the expansion and validation of investigational models, including the development of NF1 zebrafish models and patient-derived organoids. NF1 tumor organoid tissues, grown in the lab, mimic the functioning of an actual organ.

Children’s National Leads the Way

In 2007, Dan and Jennifer Gilbert established The Gilbert Family Neurofibromatosis Institute at Children’s National. The Institute advances basic and clinical research, providing a global destination for care. The Gilbert family endowed three professorships that help the hospital advance the field.

Previous funding from the Gilbert Family Foundation has improved our understanding of NF1-related gliomas and how best to treat them. It has supported pioneering work in evaluating the safety and cognitive benefits of novel treatments, primarily MEK inhibitors. The Foundation’s new award will build upon this work. It will yield new insights into one of the most lethal manifestations of NF1: transformation of low-grade gliomas into malignant lesions. Children’s National will explore novel therapies, including immunotherapy, with the potential to save lives.

Gilbert Family Neurofibromatosis Institute accomplishments include:

Children’s National Hospital once again ranked among the nation’s best by U.S. News & World Report

US News badgesChildren’s National Hospital in Washington, D.C., was ranked as a top hospital in the nation by the U.S. News & World Report 2025-26 Best Children’s Hospitals annual rankings. This marks the ninth straight year Children’s National has made the Honor Roll list. The Honor Roll is a distinction awarded to only 10 children’s hospitals nationwide.

For the fifteenth straight year, Children’s National ranked in 10 specialty services and is the highest U.S. News ranked children’s hospital in Washington, D.C., Maryland and Virginia. Last year, U.S. News introduced pediatric & adolescent behavioral health as a service line in its rankings. While there are no ordinal rankings for behavioral health, the Children’s National program was named one of the top 50 programs in the country for the second year in a row.

“To be named among the nation’s top children’s hospitals for nine years in a row is a reflection of the extraordinary expertise, innovation and heart that our teams bring to every child and family we serve,” said Michelle Riley-Brown, MHA, FACHE, president and chief executive officer of Children’s National. “Our leadership in specialties like neurology, cancer, and diabetes and endocrinology underscores the national impact of our work, and we remain focused on setting new standards in pediatric care.”

The annual rankings are the most comprehensive source of quality-related information on U.S. pediatric hospitals and recognizes the nation’s top 50 pediatric hospitals based on a scoring system developed by U.S. News.

“Being a top-ranked pediatric hospital means more than just excelling in a single specialty — it means being a pillar of outstanding care for your entire region,” said Ben Harder, chief of health analysis and managing editor at U.S. News. “Our rankings acknowledge these hospitals for their comprehensive excellence, helping families find the very best care conveniently located within their state and community.”

The bulk of the score for each specialty service is based on quality and outcomes data. The process includes a survey of relevant specialists across the country, who are asked to list hospitals they believe provide the best care for patients with the most complex conditions.

The Children’s National specialty services that U.S. News ranked in the top 10 nationally are:

The other six specialties ranked among the top 50 are Behavioral Health, Cardiology and Heart Surgery, Gastroenterology and GI Surgery, Neonatology, Pulmonology and Lung Surgery, and Urology.

Children’s National experts at the Child Neurology Society 2025 Annual Meeting

Child Neurology Society (CNS) Annual Meeting logoPhysicians, fellows and faculty from the Neuroscience and Behavioral Medicine Center at Children’s National Hospital will participate in this year’s Child Neurology Society (CNS) Annual Meeting, held October 8–11 in Charlotte, North Carolina. This premier gathering offers child neurologists the opportunity to exchange knowledge, foster collaboration, share research and advance patient care in the field.

Experts from Children’s National will be featured throughout the program, showcasing their leadership and expertise across a range of sessions and presentations.

Speakers and topics

Wei-Liang Chen MD
Seminar 9: Games Neurologists Play: AI for Enhanced Diagnosis in Neurology – Brain2Gene – Next Generation Phenotyping Using Artificial Intelligence to Predict the Genetic Diagnosis by Brain MRI Analysis
Friday, October 10, 2025 – 9:30-10:45 AM EDT/EST

Marc DiSabella, DO
Seminar 8: The Evolution of Migraine Disease from Childhood to Young Adult
Friday, October 10, 2025 – 9:30-10:45 AM EDT/EST

Hannah Grindling, MMSc, GC
CNS Genetics Summit: Demystifying Next-generation Sequencing: A Hands-On Workshop for Pediatric Neurologists
Saturday, October 11, 2025 – 12:30-4:30 PM EDT/EST

Dana Harrar MD, PhD
Seminar 7: Optimizing NeuroICU Care: Treating Patients Rather Than Seizures – Novel Therapies and Thresholds
Friday, October 10, 2025 – 9:30-10:45 AM EDT/EST

Ilana L Kahn, MD
Seminar 2: Updates in Pediatric Multiple Sclerosis
Thursday, October 9, 2025 – 2:00-3:15 PM EDT/EST

Alexandra B. Kornbluh, MD
Seminar 2: Updates in Pediatric Multiple Sclerosis – Updates in MS Diagnostic Criteria and Implications for Pediatric MS Treatment
Thursday, October 9, 2025 – 2:00-3:15 PM EDT/EST

Juma Shee Mbwana, MD
Symposium III: Year in Review Symposium – Global Pediatric Neurology
Saturday, October 11, 2025 – 9:00 AM-12:00 PM EDT/EST

John Schreiber, MD
CNS Genetics Summit: Demystifying Next-generation Sequencing: A Hands-On Workshop for Pediatric Neurologists
Saturday, October 11, 2025 – 12:30-4:30 PM EDT/EST

Kuntal Sen, MD
Workshop 7: Discovering Mentors: Early Career Leadership Guidance
Wednesday, October 8, 2025 – 3:00-4:00 PM EDT/EST
CNS Genetics Summit: Demystifying Next-generation Sequencing: A Hands-On Workshop for Pediatric Neurologists
Saturday, October 11, 2025 – 12:30-4:30 PM EDT/EST

Jeffrey Strelzik, MD
Seminar 8: The Evolution of Migraine Disease from Childhood to Young Adult – Migraine Disease in Early Childhood
Friday, October 10, 2025 – 9:30-10:45 AM EDT/EST
CNS Genetics Summit: Demystifying Next-generation Sequencing: A Hands-On Workshop for Pediatric Neurologists
Saturday, October 11, 2025 – 12:30-4:30 PM EDT/EST

Sinan Turnacioglu, MD
Seminar 9: Games Neurologists Play: AI for Enhanced Diagnosis in Neurology
Friday, October 10, 2025 – 9:30-10:45 AM EDT/EST

Poster and abstract presentations

  • From Bedside to Smartphone: Standardizing Pediatric Seizure Discharge Education Through Digital Access, Ilyse Genser, MD, Anne Vasiliadis, NP, CPNP-AC, Lauren Vilardo, MD
  • Spectrum of Neurofascin-155 Antibody Mediated Paranodopathy in a Pediatric Cohort: A Single Center Experience, Allison Saunders, MD

Additionally, Dr. DiSabella will be honored with the prestigious Program Director Award, recognizing his outstanding leadership in education, mentorship and curriculum development within child neurology and neurodevelopmental pediatrics.

For more information and to view the full schedule, please visit the CNS conference website.

A new electroencephalogram (EEG) measure may help track brain growth in Angelman syndrome

A pair of hands holding a cutout of a head

A joint study from Children’s National and UCLA findound that peak alpha frequency (PAF), a simple EEG signal, may help track brain growth in Angelman syndrome.

Angelman syndrome (AS) affects how a child’s brain develops and communicates. Families often face limited or absent speech, seizures, sleep problems and global delays. As several treatments move through late-stage trials, teams need clear ways to see if a therapy is changing the brain. A joint study from Children’s National Hospital and the University of California, Los Angeles (UCLA) points to a simple signal from an electroencephalogram (EEG), a test that records brain activity through small sensors on the scalp.

The signal is called peak alpha frequency (PAF). Alpha is a natural brain rhythm. In most children, the speed of this rhythm rises through childhood as networks mature. The study shows that this pattern is different in Angelman syndrome. Many children with AS have no clear alpha peak. When a peak is present, it is slower than in peers and does not speed up with age. That pattern suggests stalled or altered maturation of brain circuits.

Researchers looked at hundreds of EEGs from children with AS and from age-matched peers. They measured alpha activity in two independent ways to make sure the results were not due to a single analysis choice. Both approaches told the same story. In children who are typically developing, more than nine out of ten EEGs showed a distinct alpha peak, and that peak tended to get faster with age. In children with AS, fewer than half of EEGs showed a peak. When a peak appeared, it sat at a lower frequency and stayed flat across ages.

The team also confirmed a second feature of Angelman EEGs that others have reported: more slow-wave (delta) activity and less overall alpha power than peers. Taken together, these signals point to broad differences in how circuits work in Angelman syndrome.

What could this mean for care and for trials? EEG is familiar, safe, and widely available. If a therapy begins to change how circuits function, that shift could show up in PAF before day-to-day behavior changes are obvious. Adding PAF to trial measures could give clinicians and families an earlier read on brain effects. It can also sit alongside delta power to give a fuller picture – delta reflects broad disruption, while PAF tracks the speed of a key rhythm tied to growth.

There are limits to keep in mind. The EEGs came from several sites and systems, and some recordings were longer than others. The team accounted for these factors in their models, but future work with unified setups will help. A major next step is to follow the same children over time to see whether PAF changes within a child and how those changes relate to speech, movement, seizures, or sleep. The study also hints that genetic subtypes may differ, which could sharpen how trials are designed.

This project was a true collaboration between Children’s National Hospital (PI and corresponding author: Michael S. Sidorov, PhD, co-first author: Sapphire Bowen-Kauth) and UCLA (co-first author: Abigail H. Dickinson, PhD), with contributions from colleagues across both centers. Funding came from the Foundation for Angelman Syndrome Therapeutics (FAST).

For families, the takeaway is hopeful. A routine test can capture a signal linked to brain growth. In Angelman syndrome, that signal is often missing or slow, but it can be measured and tracked. As trials continue, PAF may help teams spot early signs that a therapy is reaching the brain’s core rhythms, information that matters for decisions in clinics and for progress in research.

Read the full study, “Atypical alpha oscillatory EEG dynamics in children with Angelman syndrome,”  in Neuroimage: Clinical.

Breaking Barriers: Emerging Therapies in Pediatric Brain Tumors

Dr. Matthew Bramble named Pew Scholar in Biomedical Sciences

Matthew Bramble

“This award is a tremendous honor,” said Dr. Bramble, principal investigator in the Center for Precision Medicine and Genomics Research at Children’s National. “It will allow us to push the boundaries of our work and explore new ways to understand and treat devastating childhood diseases.”

Children’s National Hospital is proud to congratulate Matthew Bramble, PhD, on being named a 2025 Pew Scholar in the Biomedical Sciences. This highly competitive honor recognizes outstanding early-career researchers who are driving innovation and discovery in human health. Dr. Bramble is one of just 22 scientists nationwide selected this year to join the Pew Scholars Program, which provides four years of funding and access to a network of more than 1,000 leading biomedical researchers.

“This award is a tremendous honor,” said Dr. Bramble, principal investigator in the Center for Precision Medicine and Genomics Research at Children’s National. “It will allow us to push the boundaries of our work and explore new ways to understand and treat devastating childhood diseases.”

Dive deeper

Dr. Bramble’s research focuses on konzo, a little-known but serious neurological condition that causes sudden and irreversible paralysis in children. Konzo primarily affects families in rural regions of sub-Saharan Africa who rely on improperly processed cassava as a staple food. The disease is linked to compounds in cassava that become toxic when combined with poor nutrition.

“Konzo is heartbreaking because it strikes vulnerable children and is entirely preventable,” said Dr. Bramble. “If we can better understand the molecular pathways that lead to paralysis, we can develop strategies to protect children and potentially inform treatments for other neurological disorders.”

His lab uses advanced OMIC’s technologies to study how toxins from cassava interact with a child’s biology, aiming to uncover how the disease damages the nervous system. By examining the molecular biology of konzo, Dr. Bramble hopes to reveal insights not only for affected communities abroad but also for understanding broader principles of brain health and development.

Why it matters

The Pew Scholars Program in the Biomedical Sciences, run by The Pew Charitable Trusts, has supported early-career scientists for 40 years. Scholars receive multiyear funding to pursue bold, creative approaches to complex scientific problems. Many alumni have gone on to win major scientific awards and make discoveries that have shaped modern medicine.

For Dr. Bramble, joining the program provides vital support for high-risk, high-reward science and connects him to a collaborative network of researchers across disciplines.

“This award validates the importance of studying conditions like konzo that don’t often receive attention but have a profound impact on children’s lives,” he said.

Dr. Bramble’s recognition underscores not only Children’s National Hospital’s commitment to advancing pediatric research and fostering discoveries that improve children’s health worldwide but also the institution’s commitment to providing resources and a setting for this type of unique, cutting-edge work. By supporting investigators who explore fundamental biology and rare diseases, the hospital helps build the foundation for tomorrow’s treatments.

With the support of the Pew award, Dr. Bramble and his team are poised to deepen their understanding of konzo and its impact on the nervous system—and, in the process, generate insights with far-reaching implications for pediatric neurology.

Marc DiSabella, DO, honored with Child Neurology Society 2025 Training Director Award

Marc DiSabella, DO

Marc DiSabella, DO, of Children’s National Hospital has been named the 2025 recipient of the Child Neurology Society (CNS) Training Director Award.

Marc DiSabella, DO, of Children’s National Hospital has been named the 2025 recipient of the Child Neurology Society (CNS) Training Director Award. This national recognition honors program directors who demonstrate outstanding leadership in education, mentorship and curriculum development within child neurology and neurodevelopmental pediatrics.

Dr. DiSabella joined Children’s National and 2008 and currently serves as associate chief of Neurology Operations, director of the Headache Program.  He previously led the Child Neurology Residency Program and served as director of Neurology Education.

A dedicated educator, Dr. DiSabella completed the Master Teacher Leadership Development Program at the George Washington University. His innovative teaching style and commitment to excellence have helped raise the bar for neurology education at Children’s National and beyond.

“Dr. DiSabella is an exceptional, enthusiastic and lucid teacher who has introduced several successful innovations over the past 15 years,” said William D. Gaillard, MD, Chief of Neurology at Children’s National. “He has trained more than 50 child neurologists, many now leaders in the field, and built one of the country’s foremost multidisciplinary headache programs. His administrative talents and positive, can-do attitude continue to benefit the entire department.”

Dr. DiSabella will be honored during the CNS Annual Meeting in Charlotte, North Carolina, in October.

The CNS Training Director Award reflects the organization’s mission to recognize the achievements of child neurologists  who are model educators, and to recognize those who shape the future of child neurology through education, mentorship and leadership.

New advances in AI help doctors understand pediatric brain tumors

person studying brain scans

One of the first and most important steps in fighting brain tumors is to understand exactly where they are and how big they are – using magnetic resonance imaging (MRI) scans.

Brain tumors are the most common cause of cancer-related death in children. These tumors can be very difficult to treat because they often grow in very delicate areas of the brain. One of the first and most important steps in fighting these tumors is to understand exactly where they are and how big they are – using magnetic resonance imaging (MRI) scans.

Traditionally, doctors have had to look at these scans and manually draw outlines around the tumors. This takes a lot of time and can vary from one doctor to another. But what if artificial intelligence (AI) could help make this easier and more efficient?

A recent international research effort called BraTS-PEDs 2023 (short for Brain Tumor Segmentation in Pediatrics) explored exactly that. This challenge co-led by Children’s National Hospital and Children’s Hospital of Philadelphia invited research teams from around the world – including a team led by Marius George Linguraru, DPhil, MA, MSc – to develop the best AI tools for automatically finding and measuring brain tumors in children’s MRIs.

A global AI competition for pediatric care

The BraTS-PEDs 2023 challenge was the first of its kind focused specifically on children. Teams were given MRI scans from 167 pediatric patients with brain tumors collected by leading consortia – the Children’s Brain Tumor Network, DMG/DIPG Registry – and Collaborative Network for Neuro-oncology Clinical Trials and reviewed by volunteer neuroradiologists from the American Society of Neuroradiology. They used these scans to train their AI programs and then tested them on new cases to see how well they performed. Our team’s algorithm was the best at measuring these tumors.

Most teams used a special type of AI network called a “U-Net,” which is great at looking at images and figuring out shapes and boundaries. The challenge showed that AI can do a very good job at finding the tumor and measuring its volume. However, it was more difficult for AI to accurately mark the small, active parts inside the tumor, known as “enhancing tumor” regions.

These results are a huge step forward. Using AI can help doctors make faster, more precise decisions, and reduce differences between hospitals or between individual doctors.

Why this matters

When doctors can accurately map out a brain tumor, they can plan surgery better, target radiation therapy more precisely, and track how well treatments are working over time. This can lead to fewer side effects and better outcomes for children.

In some cases, timely and accurate measurements can even be the difference between life and death. Having advanced AI tools could mean that children get the right treatments faster and with more confidence.

Looking ahead

The research community is now working on including even more data from different hospitals around the world and making the data and algorithm public, like Children’s National has done here. They also plan to study more types of brain tumors and scans taken after surgery and treatments. In the future, AI could become a regular part of how doctors look at brain tumors — like an extra set of smart eyes that never get tired.

At Children’s National, leaders like Dr. Linguraru are helping turn this vision into reality, giving children with brain tumors a better chance at a healthy future. You can read the full study – BraTS-PEDs: Results of the Multi-Consortium International Pediatric Brain Tumor Segmentation Challenge 2023 – in the MELBA Journal.

Other authors from Children’s National include Roger J. Packer, MD, Brian Rood, MD, Miriam Bornhorst, MD, Xinyang Liu, PhD, Zhifan Jiang, PhD, and Syed Muhammad Anwar, PhD, MS.

Transarterial embolization offers hope for infants with hemimegalencephaly

illustration of brain with brainwaves

A new study from Children’s National Hospital reveals how a minimally invasive procedure is transforming care for infants with hemimegalencephaly and medically refractory seizures, offering seizure relief with fewer surgical risks.

Until recently, families of infants with hemimegalencephaly and medically refractory seizures had few treatment options, almost all involved major brain surgery such as hemispherectomy, to remove or disconnect the affected portion of the brain. These procedures, while sometimes effective, carry high risks in very young infants due to the fragility of their developing brains and invasive nature of the surgery.

Now, specialists from the Hemimegalencephaly Program at Children’s National Hospital have pioneered a minimally invasive alternative. Leveraging the combined expertise of a multidisciplinary team — spanning neurology, neurointerventional radiology, prenatal pediatrics, neonatology, neurogenetics and neurodevelopmental pediatrics, neuropsychology and neurosurgery — they have developed a technique that induces controlled, targeted strokes in the affected hemisphere using transarterial embolization (TAE). This approach mimics the effect of surgery by reducing seizure activity, without the need for open brain procedures.

This novel strategy is the focus of a new study led by Monica Pearl, MD, director of the Neurointerventional Radiology Program. The research represents the largest retrospective review of its kind, examining 41 TAE procedures performed over an 11-year span in 13 infants under three months of age.

The results are promising: 73% of patients achieved Engel Class I seizure outcomes at follow-up, suggesting that with careful technique and patient selection, TAE may provide meaningful seizure control with fewer perioperative risks than traditional surgery. The study also highlights the technical complexity of the procedure, early complications and the critical role of evolving protocols in ensuring patient safety.

Authors from Children’s National include Monica Pearl, MD, Tayyba Anwar, MD, Shani Israel, BS, Lindsay Ruffini, CPNP, CPNP-AC, CPNP-PC, Panagiotis Kratimenos, MD, PhD, Kyle Spagnolo, DO, Wei-Liang Chen, MD, Madison Berl, PhD, William D. Gaillard, MD, Tammy N. Tsuchida, MD, PhD and Chima Oluigbo, MD

To learn more about the clinical outcomes and evolving protocols behind this approach, you can read the full study, Transarterial embolization for infants under 3 months of age with refractory seizures due to hemimegalencephaly: complication analysis and evolution of treatment strategy, in the Journal of Neurointerventional Surgery.

New AI platform accelerates brain inflammation research 10,000-fold

illustration of neurons

A new AI tool developed by Children’s National and Howard University analyzes brain immune cells 10,000x faster than manual methods.

A new Machine Learning and Artificial Intelligence tool from researchers at Children’s National Hospital (CNH) and Howard University (HU) accelerates discoveries in brain inflammation. Called StainAI, it rapidly and accurately analyzes microglia, the brain’s immune cells. Scientists currently analyze microglia slowly by hand. StainAI automates this process and speeds it up 10,000-fold. Its use will aid discovery of new treatments for inflammatory brain conditions such as infection, autoimmunity, and aging.

Solving a problem

Traditionally, scientists study microglia one cell at a time. They reconstruct each cell’s shape by hand under a microscope. The shape helps classify microglia as “resting” (normal) or “activated” (inflamed). The manual process is tedious and slow. It limits analyses to a few microglia in small brain areas.

StainAI changes that. It uses deep machine learning and artificial intelligence to overcome and exceed the manual method’s limitations. It correctly classifies millions of microglia from standard microscopic images. StainAI also localizes each microglia to its brain region in 3D. These features enable single-cell analyses of immune activity at a scale not feasible before – the entire brain.

A tool with broad impact

The team applied StainAI to two models of brain injury and inflammation to show its utility. In a rodent model of pediatric cardiac arrest, StainAI identified new brain regions susceptible to injury. In a simian model of viral infection, StainAI localized rod-shaped microglia normally found in white matter to an unexpected brain region – the hippocampus.  These findings point towards new treatments and highlight StainAI’s value across diseases and species.

StainAI is fast, accurate and adaptable. It uses common laboratory equipment. Its creators, Michael Shoykhet, MD, PhD, at CNH and Dr. Tsang-Wei Tu at HU, are making StainAI available to other researchers. They hope StainAI will help labs worldwide discover new ways to protect children’s brains from inflammation and injury.

You can read the full article, StainAI: quantitative mapping of stained microglia and insights into brain-wide neuroinflammation and therapeutic effects in cardiac arrest, in Communications Biology, a Nature group journal.

Children’s National co-leads efforts to increase skin-to-skin care for babies with congenital heart disease

The Children's National Heart Center team

The Children’s National Heart Center team led activities designed to encourage skin-to-skin contact between parents and infants in the Cardiac Intensive Care Unit and Heart and Kidney Unit.

Clinicians at Children’s National Hospital and Children’s Hospital Orange County are leading a nationwide event to encourage families to practice more skin-to-skin, or kangaroo, care with newborn infants who have congenital heart disease, including throughout hospitalization.

Thirty-one hospitals across the United States will participate in this congenital heart disease focused “Skin-to-skin-a-thon,” that will include family and clinical care provider activities and education throughout pediatric cardiac intensive care units and step-down units.

The event will celebrate the tremendous benefits that research shows both families and infants gain from physical contact early in life.

Early skin-to-skin care has been shown to:

  • Reduce stress in both baby and the parent
  • Help with baby’s physiologic stability including regulating vital signs like temperature, heart rate, and blood pressure
  • Provide infant pain relief
  • Improve infant digestion and weight gain
  • Support good sleep/wake cycles in babies.
  • Increase oxytocin for mothers, which can help improve milk production/support breastfeeding

Most studies showing these benefits have included pre-term babies or those born after a healthy term. The idea of encouraging family skin-to-skin care in the hospital setting has been widely adopted in neonatal intensive care units but is not done routinely in cardiac intensive care units. One study estimated that only 6% of parents whose babies were hospitalized for congenital heart disease reported any skin-to-skin care during their stay, with most stays averaging 22 days.

“Research shows so many benefits for all infants and their parents — and our congenital heart newborns stand to gain even more from this type of contact, but often receive it far less,” says Sarah Schlatterer, MD, PhD, medical director of Neurocardiac Critical Care at Children’s National. “This awareness effort is designed to help families understand how to do this safely and also empower our bedside care providers to encourage skin-to-skin care as much as they can every day.”

The event overall is inspired and supported by the Cardiac Newborn Neurodevelopmental Network SIG of the Cardiac Neurodevelopmental Outcomes Collaborative, who planted the seed of the idea and assisted with dissemination of information and coordinating between participating hospitals.

Stroke alert teams beneficial to timely diagnosis of pediatric stroke patients

Key driver diagram

1 – Interdisciplinary stroke simulations were canceled due to short staffing and insufficient nursing support during COVID-19 pandemic.

Diagnosis times improved for pediatric arterial ischemic stroke (AIS) patients in the emergency department after the implementation of a quality improvement initiative. According to a new study, published in Pediatrics, the median door-to-imaging time for children with suspected AIS improved from 128 minutes to 68 minutes post-intervention.

What it means

Pediatric AIS is an important cause of morbidity and mortality that requires early recognition to benefit from hyperacute therapies. A quality improvement study at Children’s National Hospital aimed to improve timely diagnosis of AIS through an interdisciplinary stroke response protocol. The researchers implemented a quality improvement initiative from November 2019 to June 2023 in the emergency department that included interventions under two categories – workflow efficiency improvement and staff education. A total of 71 patients who met the criteria for the study were analyzed. Median door-to-imaging time for all patients improved from 128 minutes during the baseline period to 68 minutes.

The researchers note that establishing a well-functioning stroke response protocol is a critical step in detecting pediatric stroke in the community and expediting care for this vulnerable patient population.

“This study highlights the value of quality improvement methodologies in coordinating multiple hospital divisions for the greater goal of improving patient care,” says Theodore Trigylidas, MD, corresponding author and emergency medicine physician at Children’s National. “We hope it serves as a template for other healthcare organizations in developing their own pediatric stroke protocols.”

Code Stroke management algorithm

Code Stroke management algorithm. CT/CTA, computed tomography with or without angiography. MRI/MRA, magnetic resonance imaging with or without angiography.

The hold up in the field

The rarity of AIS in pediatric patients and stroke mimics, like hemiplegic migraine, may cause challenges for a timely diagnosis, and treatments for AIS – thrombolysis and thrombectomy – must be performed within specific time frames.

Moving the field forward

“By taking into consideration local staffing structures and institutional resource availability, the quality improvement approach described here can serve as a framework for how institutions might implement a pediatric acute stroke response protocol given their own unique systems of care and resource considerations,” says Dana Harrar, MD, PhD, study author and director, Pediatric Stroke Program at Children’s National.

The authors plan to expand this initiative to incorporate children at outside facilities with the goal of creating a stroke network with nearby hospitals.

Authors from Children’s National include: Theodore Trigylidas, MD, Nichole McCollum, MD, Kathleen Brown, MD, Paola Pergami, MD, Elizabeth Wells, MD, Jonathan Murnick, MD, PhD, Josh Heffren, PharmD, BCPPS and Deborah LaViolette, BSN, Dana Harrar, MD, PhD.

You can read the full study, Improving Timely Diagnosis of Arterial Ischemic Stroke at the Pediatric Emergency Department in Pediatrics.

REI Week 2025 empowers the future in pediatric research and innovation

Children’s National Hospital hosted its fifteenth annual Research, Education and Innovation Week from March 31–April 4, 2025, bringing together clinicians, scientists, educators and innovators from across the institution to celebrate discovery and collaboration. This year’s theme, “Empowering the Future in Pediatric Research and Innovation with Equity, Technology and a Global Reach,” served as a call to action for advancing science that improves child health both locally and around the world.

Each day of the week-long event featured thought-provoking lectures — now available to watch — dynamic panel discussions, interactive workshops and vibrant poster sessions, all highlighting the diverse and interdisciplinary work taking place across Children’s National.

Centering the patient and the planet

REI Week began on Monday with a powerful keynote lecture from Lynn R. Goldman, MD, MS, MPH, Michael and Lori Milken dean of the Milken Institute School of Public Health at the George Washington University. In her talk, “Children: Uniquely vulnerable to climate-related threats,” Dr. Goldman underscored the urgent need to protect children from the environmental hazards of a changing climate and to integrate climate science into pediatric care and advocacy.

At mid-morning, Mary-Anne “Annie” Hartley, MD, PhD, MPH, director of the LiGHT Laboratory at École Polytechnique Fédérale de Lausanne, introduced the “MOOVE” platform — Massive Open Online Validation and Evaluation of clinical LLMs. Her talk demonstrated how artificial intelligence, when rigorously validated, has the potential to transform clinical decision-making and global health equity.

Monday’s final keynote, “Zinc and childhood diarrhea,” was presented by Christopher Duggan, MD, MPH, director of the Division of Nutrition at Harvard Medical School. Dr. Duggan highlighted the global health impact of zinc supplementation in reducing childhood mortality — a reminder that simple, evidence-based interventions can save millions of lives.

In that first day, the first poster session of the week showcased projects in adolescent medicine, global health, infectious diseases, oncology and more. The session reflected the full breadth of research taking place across Children’s National.

Ambroise Wonkam, MD, PhD, professor of genetic medicine at Johns Hopkins University, then delivered Tuesday’s Global Health Keynote Lecture, “Harnessing our common African genomes to improve health and equity globally.” His work affirmed that inclusive genomics is key to building a healthier world.

Later, the Global Health Initiative event and GCAF Faculty Seminar encouraged attendees to pursue collaborative opportunities at home and abroad, reflecting the growing global footprint of Children’s National research programs.

Transforming education and care delivery

On Wednesday, Larrie Greenberg, MD, professor emeritus of pediatrics, kicked off the day with a Grand Rounds keynote on educational transformation: “Shouldn’t teachers be more collaborative with their learners?” He followed with a CAPE workshop exploring the effectiveness of case-based learning.

The Nursing Sponsored Keynote Lecture by Vincent Guilamo-Ramos, PhD, MPH, LCSW, ANP-BC, PMHNP-BC, FAAN, explored “Redesigning the U.S. broken health system.” He offered an urgent and inspiring call to reimagine pediatric care by addressing social determinants of health.

In the Jill Joseph Grand Rounds Lecture, Deena J. Chisolm, PhD, director of the Center for Child Health Equity at Nationwide Children’s Hospital, challenged attendees to move beyond dialogue into action in her talk, “Health equity: A scream to a whisper?,” reminding researchers and clinicians that advocacy and equity must be foundational to care.

The day continued with a poster session spotlighting medical education, neonatology, urology and neuroscience, among other fields.

Posters and pathways to progress

Throughout the week, poster sessions highlighted cutting-edge work across dozens of pediatric disciplines. These sessions gave attendees the opportunity to engage directly with investigators and reflect on the shared mission of discovery across multiple disciplines, including:

Honoring excellence across Children’s National

The REI Week 2025 Awards Ceremony celebrated outstanding contributions in research, mentorship, education and innovation. The winners in each category were:

POSTER SESSION AWARDS

Basic & Translational Research

Faculty:  Benjamin Liu, PhD

“Genetic Conservation and Diversity of SARS-CoV-2 Envelope Gene Across Variants of Concern”

Faculty:  Steve Hui, PhD
“Brain Metabolites in Neonates of Mothers with COVID-19 Infection During Pregnancy”

Faculty: Raj Shekhar, PhD
“StrepApp: Deep Learning-Based Identification of Group A Streptococcal (GAS) Pharyngitis”

Post docs/Fellows/Residents: Dae-young Kim, PhD
“mhGPT: A Lightweight Domain-Specific Language Model for Mental Health Analysis”

Post docs/Fellows/Residents: Leandros Boukas, MD, PhD
“De Novo Variant Identification From Duo Long-Read Sequencing: Improving Equitable Variant Interpretation for Diverse Family Structures”

Staff: Naseem Maghzian
“Adoptive T Lymphocyte Administration for Chronic Norovirus Treatment in Immunocompromised Hosts (ATLANTIC)”

Graduate Students: Abigail Haffey
“Synergistic Integration of TCR and CAR T Cell Platforms for Enhanced Adoptive Immunotherapy in Brain Tumors”

High School/Undergraduate Students: Medha Pappula
“An ADHD Diagnostic Interface Based on EEG Spectrograms and Deep Learning Techniques”

Clinical Research

Faculty: Folasade Ogunlesi, MD
“Poor Air Quality in Sub-Saharan Africa is Associated with Increase Health Care Utilization for Pain in Sickle Cell   Disease Patients”

Faculty: Ayman Saleh, MD
“Growth Parameters and Treatment Approaches in Pediatric ADHD: Examining Differences Across Race”

Post docs/Fellows/Residents: Nicholas Dimenstein, MD, MPH
“Pre-Exposure Prophylaxis (PrEP) Eligibility in the Pediatric Emergency Department”

Staff: Tayla Smith, MPH
“The Public Health Impact of State-Level Abortion and    Firearm Laws on Health Outcomes”

Graduate Students: Natalie Ewing
“Patterns of Bacteriuria and Antimicrobial Resistance in Patients Presenting for Primary Cloacal Repair: Is Assisted Bladder Emptying Associated with Bacteriuria?”

Graduate Students: Manuela Iglesias, MS
“Exploring the Relationship Between Child Opportunity Index and Bayley-III Scores in Young Children”

High School/Undergraduate Students: Nicholas Lohman
“Preliminary Findings: The Efficacy, Feasibility and Acceptability of Group Videoconference Cognitive Behavioral Therapy with Exposure and Response Prevention for Treating Obsessive-Compulsive Disorder Among Children and Young People”

Community-Based Research

Faculty: Sharon Shih, PhD
“Assessing Pediatric Behavioral Health Access in DC using Secret Shopper Methodology”

Post docs/Fellows/Residents: Georgios Sanidas, MD
“Arrested Neuronal Maturation and Development in the Cerebellum of Preterm Infants”

Staff: Sanam Parwani

“Intersectionality of Gender and Sexuality Diversity in Autistic and Non-Autistic Individuals”

Graduate Student: Margaret Dearey
“Assessing the Burden of Period Poverty for Youth and Adolescents in Washington, DC: A Pilot Study”

Quality and Performance Improvement

Faculty: Nichole L. McCollum, MD
“A Quality Improvement Study to Increase   Nurse Initiated Care from Triage and Improve Timeliness to Care”

Post docs/Fellows/Residents:  Hannah Rodriguez, MD
“Reducing Unnecessary Antibiotic Use in a Level IV NICU”

Staff: Amber K. Shojaie, OTD, OTR/L
“Implementing Dynamic Axilla Splints in a Large Burn Patient”

MENTORSHIP AWARDS

Basic Science Research

Conrad Russell Y. Cruz, MD, PhD

Clinical Research

Rana Hamdy, MD, MPH, MSCE

Bench to Bedside Research

Ioannis Koutroulis, MD, PhD, MBA

ELDA ARCE TEACHING SCHOLAR AWARD

Priti D. Bhansali, MD, MEd

Heather Ann Walsh, PhD, RN

SUZANNE FEETHAM NURSING RESEARCH SUPPORT AWARD

Eileen P. Engh, PhD RN 
“Rare Disease Organization Lifecycle” Role in Helping Parents with Everyday Life Information Seeking and Connection (RDO-HELIX)

EXPLORATIONS IN GLOBAL HEALTH PILOT AWARDS

Launchpad Awards

Mi Ran Shin, MD, MPH
“Establishing Interdisciplinary Rehabilitation for Birth and Burn Injuries in Ethiopia”

Susan Harvey, MSN, CPNP-AC
“Implementation of Sickle Cell Pilot Program in Ndhiwa Sub County, Kenya”

Meleah Boyle, PhD, MPH
“Understanding and Addressing Environmental Sustainability to Protect the Health of the Children’s National and Global Communities”

Eiman Abdulrahman, MD
“Research Capacity Building to Improve Pediatric Emergency and Critical Care in Ethiopia”

Pilot Awards

Alexander Andrews, MD
“EEG as a Diagnostic and Prognostic Marker in Severe Pediatric Malaria, Blantyre Malawi”

Daniel Donoho, MD & Timothy Singer, MD
“Feasibility Study of a Novel Artificial Intelligence-Based Educational Platform to Improve Neurosurgical Operative Skills in Tanzania”

Hasan Syed, MD
“Bridging the Gap an Educational Needs Assessment for Pediatric Neurosurgery Training in Pakistan”

Sofia Perazzo, MD & Lamia Soghier, MD, MEd, MBA
“QI Mentorship to Improve Pediatric Screening and Follow-up in Rural Argentina”

Benjamin Liu, PhD
“AI-Empowered Real-Time Sequencing Assay for Rapid Detection of Schistosomiasis in Senegal”

Rae Mittal, MD
“Assessment and Enhancement of Proficiency in Emergency Child Neurology Topics for Post-Graduate Emergency Medicine Trainees in India”

Innovation Day ignites bold thinking

Thursday, REI Week shifted to the Children’s National Research & Innovation Campus for Innovation Day, a celebration of how bold ideas and collaborative culture can accelerate progress in pediatric medicine.

Brandy Salmon, PhD, associate vice president of Innovation and Partnerships at Virginia Tech, opened the day with “The Alchemy of Innovation,” focusing on how institutions can build a culture that fuels transformative partnerships.

A multidisciplinary panel discussion moderated by Nathan Kuppermann, MD, MPH, and Catherine Bollard, MBChB, MD, featured Nehal Mehta, MD, Julia Finkel, MD, Kevin Cleary, PhD, Ioannis Koutroulis, MD, PhD, MBA, Francesca Joseph, MD and Patrick Hanley, PhD, who shared how innovation can be advanced and promoted, especially as a core institutional priority.

A shared vision for the future

REI Week 2025 reaffirmed the values that define Children’s National: a commitment to excellence, collaboration and equity in pediatric research and care. As discoveries continue to emerge from our hospital and our research campuses, the connections built and ideas sparked during this week will help shape the future of pediatric health — locally and globally.

By elevating voices from the bedside to the bench, with the support of the executive sponsors Nathan Kuppermann, MD, MBChB, Catherine Bollard, MBChB, MD, Kerstin Hildebrandt, MSHS, Linda Talley, MS, RN, NE-BC and David Wessel, MD, REI Week demonstrated that we must embrace the community in all aspects of our work. Because we know that there are answers we can only get from the patients that we serve—and we need to be their voice.

Research, Education & Innovation Week will be back next year on April 13-17, 2026.

  • Posters at the REI Week 2025 Monday, March 31 poster session.

    Posters at the REI Week 2025 Monday, March 31 poster session.
  • Panelists discuss innovation during REI Week 2025.

    Panelists discuss innovation during REI Week 2025.
  • Global Health Initiative community engagement event during REI Week 2025.

    Global Health Initiative community engagement event during REI Week 2025.
  • Chris Rees presents his REI Week 2025 lecture.

    Chris Rees presents his REI Week 2025 lecture.
  • Nathan Kuppermann listens to a presenter during the REI Week 2025 Tuesday, April 1, poster session.

    Nathan Kuppermann listens to a presenter during the REI Week 2025 Tuesday, April 1, poster session.
  • Michelle Riley-Brown, Nathan Kuppermann, Catherine Bollard and Naomi Luban on stage during the REI Week 2025 awards ceremony.

    Michelle Riley-Brown, Nathan Kuppermann, Catherine Bollard and Naomi Luban on stage during the REI Week 2025 awards ceremony.
  • Brandy Salmon presents on innovation programs at Virginia Tech during the REI Week 2025 Innovation Day.

    Brandy Salmon presents on innovation programs at Virginia Tech during the REI Week 2025 Innovation Day.
  • Catherine Bollard listens to a presenter during the REI Week 2025 Monday, March 21 poster session.

    Catherine Bollard listens to a presenter during the REI Week 2025 Monday, March 21 poster session.
  • Ambroise Wonkman poses for a picture with Children’s National staff.

    Ambroise Wonkman poses for a picture with Children’s National staff.
  • Tanzeem Choudhury presenting during REI Week 2025.

    Tanzeem Choudhury presenting during REI Week 2025.

Pioneering NF innovation and treatment: Q&A with Benjamin Siegel, MD

Benjamin Siegel, MDFor more than four decades, Children’s National Hospital has been at the forefront of neurofibromatosis (NF) care, research and treatment. In 2007, Dan and Jennifer Gilbert furthered this mission by establishing the Gilbert Family Neurofibromatosis Institute in honor of their son Nick. The Gilberts have dedicated themselves to eradicating NF and improving the future for those affected. Their partnership with Children’s National has transformed the Institute into a global hub for NF care and innovation.

“Since 1982, we’ve combined compassionate care with cutting-edge innovation to provide the best outcomes for our patients while advancing NF treatment,” says Roger J. Packer, MD, director of the institute.

Dr. Packer leads a team of NF experts, including Benjamin Siegel, MD, clinical co-director of the institute, whose expertise in pediatric neurology and neuro-oncology is critical to the program’s success. In this Q&A, Dr. Siegel shares what sets Children’s National apart, how it benefits patients and the latest NF research advancements.

What makes the Gilbert Family Neurofibromatosis Institute at Children’s National unique from other programs in the country?

The Gilbert Family Neurofibromatosis Institute is the longest-running NF program in the United States, with over 40 years of leadership in advancing the field. We helped establish the initial diagnostic criteria in the 1980s and have been pivotal in chairing the Neurofibromatosis Clinical Trials Consortium. Our involvement in groundbreaking clinical trials led to FDA approval of the first targeted therapies for NF1-associated plexiform neurofibromas. Building upon this strong legacy, we continue to advance the future of care for those affected by NF.

Our multidisciplinary team, led by Sinan Turnacioglu, MD, and myself under Dr. Packer’s direction, integrates diverse expertise. My background in neurology and neuro-oncology, combined with Dr. Turnacioglu’s expertise in neurodevelopmental disabilities, allows us to provide comprehensive, high-quality care. With access to specialized clinical resources, we ensure each patient receives the most informed and well-rounded treatment.

How does the work in this program benefit patients?

Our program’s success lies in its comprehensive, interdisciplinary approach to patient care. NF and schwannomatosis can impact every organ system in the body, making close collaboration among specialists essential to address each patient’s unique needs. Our goal is to act as a “medical home” for individuals with NF, ensuring seamless, coordinated care across neurology, oncology, genetics, orthopedics, neurosurgery, plastic surgery, otolaryngology, endocrinology, cardiology and psychiatry.

Recent expansions include neuropsychology, led by Karin Walsh, PsyD, providing comprehensive clinical neuropsychological testing, and physical medicine and rehabilitation, where Mi Ran Shin, MD, MPH, helps manage musculoskeletal issues and chronic pain. This integrated approach allows us to address the complex concerns of NF patients effectively, improving both their physical function and quality of life.

How is Children’s National leading the way in NF research?

Children’s National is at the forefront of NF research, with groundbreaking translational studies spearheaded by its cutting-edge hospital laboratories. Our team is pioneering the use of organoid models to better understand malignant transformations in NF1-associated low-grade gliomas. With a focus on developing targeted therapies for currently untreatable high-grade gliomas, Children’s National has been selected as a key member of the newly funded Gilbert Family NF1-Assosiated Glioma Consortium, an initiative dedicated to advancing treatment options for NF1-associated gliomas.

As a founding member of the Neurofibromatosis Clinical Trials Consortium (NFCTC), we led pivotal clinical trials on targeted therapies for NF1 and schwannomatosis (NF2) tumors. In 2025, the consortium will launch three new trials focused on NF1-associated plexiform neurofibromas. In development are trials evaluating bone health in NF1, attention deficit disorder in NF1, preventative therapy for NF2-associated schwannomas and new treatments for high-grade gliomas and malignant peripheral nerve sheath tumors (MPNST).

Beyond the NFCTC, Children’s National is a member of the Pediatric Brain Tumor Consortium, contributing to selumetinib research for NF1 gliomas. Additionally, through the leadership of internationally known solid tumor oncologist AeRang Kim, MD, PhD, we’re involved with the Sarcoma Alliance for Research through Collaboration, with a focus on developing treatments for MPNST.

Dr. Walsh’s neuropsychology program has advanced understanding of NF1-related cognitive challenges through studies on MEK inhibitors’ effects on neurocognition and links between sleep disorders and ADHD. It also recently completed a pilot study on the Unstuck and On Target program to improve executive function in NF1.

What else is important for peers to know?

A diagnosis of NF or schwannomatosis can be overwhelming and frightening for families due to the wide range of symptoms and manifestations. This uncertainty can often lead to significant anxiety, as patients and their families may struggle to understand what to expect in terms of medical care, progression and treatment options.

At the Gilbert Family Neurofibromatosis Institute, we aim to ease this uncertainty by providing a comprehensive care approach. We offer not only cutting-edge clinical treatments but also support and guidance through the complexities of the disorder. Our team is committed to addressing concerns, providing updated information about the latest research and connecting families to ongoing clinical trials and support resources. By offering a personalized, multi-disciplinary approach, we help our patients navigate their diagnosis with greater confidence and hope for the future.

Looking ahead, Children’s National is set to advance NF research and care with a five-year grant from the Gilbert Family Foundation. The hospital will collaborate with global institutions to address NF-1 associated transformed gliomas and launch the first clinical study using molecular targeted therapy and immunotherapy to treat this aggressive tumor, marking a key milestone in its commitment to innovative treatments and improved outcomes for children with NF.

New study finds prenatal opioid exposure linked to smaller newborn brain volumes

brain scans of newbornA new study out of the Center for Prenatal, Neonatal & Maternal Health Research, directed by Catherine Limperopoulos, PhD, led by Yao Wu, PhD and Stephanie Merhar, MD, MS, out of Cincinnati Children’s Hospital Medical Center, sought answers to the question: “Do brain volumes differ in opioid-exposed vs. unexposed newborns?” In one of the largest studies of this kind, researchers found that prenatal exposure to opioids is associated with smaller brain volumes in newborns. These findings from the landmark Outcomes of Babies with Opioid Exposure (OBOE) study build on, reinforce smaller studies, and achieve a better understanding of the impacts of prenatal opioid exposure (POE).

Dive Deeper

The Advancing Clinical Trials in Neonatal Opioid Withdrawal (ACT-NOW)’s OBOE study is a multi-site observational study of newborns with prenatal opioid exposure and a control group of unexposed newborns from four different sites in the United States – Case Western Reserve University, Cincinnati Children’s Hospital Medical Center, University of Alabama at Birmingham, and Children’s Hospital of Philadelphia.

In a study involving 173 newborns who were exposed to opioids during pregnancy and 96 newborns not exposed to opioids prenatally showed smaller brain sizes in several key areas. Specifically, these exposed babies had smaller total brain volumes, as shown through MRI, as well as reduced volumes in important parts of the brain, including the cortex (outer layer of the brain), deep gray matter (areas that control movement and emotions), white matter (which helps transmit signals in the brain), cerebellum (responsible for coordination and movement), brainstem (controls basic functions like breathing), and the amygdala (involved in emotions and memory).

Further details showed that newborns exposed to medication for opioid use disorder (MOUD) during pregnancy with methadone, had smaller white matter volumes, while those exposed to MOUD with buprenorphine had smaller volumes specifically in the right amygdala. Additionally, newborns who were exposed to opioids plus additional substances such as THC and gabapentin had smaller volumes in even more brain areas compared to those who were only exposed to opioids.

What’s Next

The OBOE study sets the groundwork for further research into the long-term impact of opioid exposure during pregnancy. Additional work is necessary to expand on these findings and how they relate to functions in childhood – including exploring the way these reduced brain volumes may impact cognitive, behavioral, and motor impairments. The study raises important questions about how current guidelines for MOUD during pregnancy – specifically with methadone and buprenorphine – might evolve considering these findings. This study highlights the need for further research to assess the long-term effects of MOUD regimens on both maternal and infant outcomes.

This significant study underscores the importance of multi-disciplinary collaboration in opioid exposure research, effective regulation, and policy interventions – involving healthcare providers, researchers, policymakers, and affected families – to best mitigate the consequences and improve the health outcomes of children affected by prenatal opioid exposure.

You can read the full study, Antenatal Opioid Exposure and Global and Regional Brain Volumes in Newborns, and its companion editorial, Following the Developing Brain Affected by Opioid Exposure, in the Journal of the American Medical Association.

Additional authors from Children’s National include Kushal Kapse, BS, MS, and Josepheen De Asis-Cruz, MD, PhD. Other authors include Carla M. Bann, PhD, Jamie E. Newman, PhD4, Nicole Mack, MS, Sara B. De Mauro, MD, MSCE, Namasivayam Ambalavanan, MD, Jonathan M. Davis, MD, Scott A. Lorch, MD, MSCE5, Deanne Wilson-Costello, MD, Brenda B. Poindexter, MD and Myriam Peralta-Carcelen, MD.

Children’s National Hospital to host 35th Annual Pediatric Neurology Update

illustration of the brain on black backgroundThe Divisions of Neurology and Neurosurgery at Children’s National Hospital are proud to host the 35th Annual Pediatric Neurology Update course.

Chair Elizabeth Wells, MD; Co-Chairs, Marc DiSabella, DO, John Schreiber, MD, William D. Gaillard, MD, Robert Keating, MD

The course attracts a national audience and brings together neuroscience clinicians and pediatricians in the Washington, D.C. and Mid-Atlantic region.

Guest speakers include Annapurna Poduri, MD, MPH, Deputy Director for NINDS, Emily Freilich, MD, from the FDA and Conor Mallucci, MBBS, Chief of Neurosurgery at Alder Hays, England.

This year’s course highlights 3 major areas:

  • Updates in Epilepsy
  • Innovations in Vascular Neurosurgery and Neurointerventional Radiology
  • Addressing Mental and Behavioral Health in Neurological Conditions

We invite you to join us for presentations from experts in the field during this full-day, CME accredited event on April 10, 2025. This is a hybrid event that will be held virtually or in-person at the Children’s National Research & Innovation Campus.

For more information and to register, visit ChildrensNational.org/NeurologyUpdate.

U.S. News & World Report voting

U.S. News & World Report badgeChildren’s National is ranked one of the top 10 pediatric hospitals in the nation by U.S. News & World Report. Our faculty and staff are proud of the impact made on the lives of children and families in our community. Your participation in the U.S. News & World Report annual reputational survey validates the quality of care we provide and reflects the mutual respect and trust we share as healthcare professionals.

How to determine your voting eligibility

Voting for the U.S. News & World Report Best Children’s Hospitals rankings can be done only through Doximity.

To participate, physicians must:

  • Be board-certified and meet the eligibility criteria for the voting categories.
  • For child and adolescent psychologists, your account must be up to date with your specialty and subspecialty correctly marked.
  • Be a credential-verified member of Doximity (you must have an active and claimed Doximity profile).
  • Have all certifications and board documents currently up-to-date in your Doximity profile.

View the full eligibility criteria

How to claim your Doximity profile to vote

  • You have to claim your profile on Doximity.com to participate in the online survey. If you have not yet claimed your Doximity profile, go to Doximity.com, and click “Find My Profile.”
  • Once your profile has been claimed, you must confirm your email address and board certifications.
  • Verified Doximity members will receive an email inviting them to participate in the U.S. News survey.
  • For more information on how to claim your profile, visit Doximity.com

How to update and verify existing Doximity account information

Your Doximity profile must have up-to-date licenses, certifications and board documents.

  • Once you are logged in, your profile will automatically be in “Edit Mode.” You are able to add new items or edit existing information.
  • Update your Doximity profile and ensure your information is current.

Once registered, users wishing to participate in the online survey should:

  • Watch for an email from Doximity about the annual member survey.
  • Even if you don’t see the email, if you are a registered Doximity user, you can still vote by logging in to Doximity.com with your username and password during the voting period.
  • Once logged in, look for a U.S. News graphic or button on the homepage and click on it.
  • The survey asks users to name the hospitals that provide the best care in your respective specialty, without consideration to location or cost. Pediatric specialists will list 10 hospitals. The order in which you list the hospitals does not matter.

Please note: Children’s National Hospital is listed as “Children’s National Hospital Washington, DC” on the survey.

Visit Doximity’s FAQs if you have issues or questions about registration or claiming your profile.

How to cast your vote

In February 2025 when voting opens, all survey-eligible physicians will receive a notification on the Doximity app for Android or iOS. If you do not use the Doximity app, you will receive an email when voting opens.

  1. Log in to your Doximity account at doximity.com or via the mobile app.
  2. Click the Notifications icon or tap the “Submit your Nominations” button on the homepage. You can also search for “U.S. News Best Hospitals”
  3. Select 10 hospitals in your respective specialty that you believe provide the best care in the United States.
  4. Submit your vote

Having technical issues?

If you have difficulty registering with Doximity or completing the survey, please visit Doximity support for assistance.

Vote

The 2025 U.S. News & World Report Best Children’s Hospitals reputation voting will open in mid-February. Look for your Doximity notification to vote.

Study finds new genetic cause of neurodevelopmental disorders

Histological section of a brain.

Nissel staining of pre-clinical model brain.

A cross-disciplinary team of researchers and physicians from Children’s National Hospital and Washington University School of Medicine in St. Louis, in collaboration with physicians from around the world, identified a new genetic cause of neurodevelopmental disorders (NDDs). In a new study published in the American Journal of Human Genetics, researchers found 14 unrelated patients with 15 different sequence variants in HECTD1 – 10 missense, 3 frameshift, 1 nonsense and 1 splicing variant – with NDDs, including autism, attention-deficit/hyperactivity disorder (ADHD) and epilepsy.

Moving the field forward

Many patients suspected of having a genetic disorder remain undiagnosed. In about 10% of these cases, the genetic change is in a gene unknown to cause the disorder.

“Describing a new genetic cause of neurodevelopmental disorders will allow for the characterization of the gene’s role in brain development, the genetic syndrome and the mechanisms of disease,” says Irene Zohn, PhD, principal investigator in the Center for Genetic Medicine Research at Children’s National and co-lead of the study. “This information will lead to developing treatments to improve the lives of patients.”

The patient benefit

“Our study represents the first report of HECTD1 in NDDs,” says Dr. Zohn. “Now that this gene is linked to the disorder, clinicians with patients with sequence variation in this gene can enroll in studies to understand this new HECTD1 syndrome.”

Proper genetic diagnosis is important so that comorbidities and the natural history of the disorder can be described, which will lead to improvements in patient care.

What we hope to discover

Now that a new genetic syndrome has been defined, researchers hope to establish how prevalent the syndrome is and describe its features. Using pre-clinical models, they’ll continue to study the developmental basis of the disorder and the molecular mechanisms to develop therapies.

Children’s National leads the way

The HECTD1 gene was discovered in Dr. Zohn’s laboratory, and her research team connected with Christina Gurnett, MD, PhD, co-lead of the study from Washington University School of Medicine, to link this gene to human disease.

Additional authors from Children’s National include – Elias Oxman, Keito Ishibashi, Sonia Sebaoui, Kathryn Luedtke, Bryce Winrow, Seth Berger, MD, PhD, Christina L. Grant, MD, PhD, and Joshua Corbin, PhD.

You can read the full study Sequence Variants in HECTD1 Result in a Variable Neurodevelopmental Disorder in the American Journal of Human Genetics.