Diabetes & Endocrinology

2025 with a lightbulb instead of a zero

The best of 2025 from Innovation District

2025 with a lightbulb instead of a zero

In 2025, Innovation District readers gravitated toward stories that explored how research and clinical innovation are reshaping pediatric care in real time. This year’s most popular articles highlighted advances in complex surgical care, evidence-based treatments for chronic and neuropsychiatric conditions and emerging technologies — from wearable data to artificial intelligence — that are changing how clinicians diagnose, treat and support children and families. Read on for our list of the most popular articles we published on Innovation District in 2025.

1. Life-changing care: How Children’s National tackles pediatric cervical spine injuries

The Cervical Spine program at Children’s National Hospital is responsible for treating a range of conditions, including trauma, congenital abnormalities and tumors. These conditions can lead to instability or misalignment of the cervical spine. “There are unique challenges in pediatric cases due to anatomical differences. The cervical spines of children are more at risk for injury because of their developmental stage and structural characteristics,” says Matthew Oetgen, MD, MBA, chief of Orthopaedic Surgery and Sports Medicine at Children’s National.
(2 min. read)

2. Pioneering evidence-based treatments for substance addictions

Increasing evidence-based treatment is a key component of the Addictions Program at Children’s National Hospital, created in 2022 and led by Sivabalaji Kaliamurthy, MD. “We really want to focus on intervening in an evidence-based manner in the primary care setting because that is where most of our patients are going to first access care outside of the emergency room,” explains Dr. Kaliamurthy.
(3 min. read)

3. Breaking barriers in growth disorder treatment for families

For many children with short stature and other rare genetic growth disorders, there have been no next steps after usual treatment options prove ineffective. Researchers at Children’s National Hospital are digging deeper to find the root genetic causes of short stature disorders and creating novel, nuanced treatment options that have the opportunity to change how the field approaches these cases.
(4 min. read)

4. The link between metabolic acidosis and cardiovascular disease in children with CKD

Denver D. Brown, MD, nephrologist at Children’s National, is looking at whether untreated metabolic acidosis could potentially contribute to cardiovascular outcomes in children with chronic kidney disease (CKD). Here, she explains her motivation, findings and future directions for this critical research.
(3 min. read)

5. Therapy approach shows promise for PANS/PANDAS

A multidisciplinary therapy model developed at Children’s National shows promise for children with PANS and PANDAS, significantly reducing symptoms through structured cognitive-behavioral therapy and family-centered care. The approach could offer a new standard for treating these rare, complex neuropsychiatric disorders.
(2 min. read)

6. Wearable tech data shows promise in ADHD detection

A study from Children’s National reveals that common wearable devices like Fitbits may hold the key to improving how we identify Attention-Deficit/Hyperactivity Disorder (ADHD) in adolescents. By analyzing patterns in heart rate, activity levels and energy expenditure, researchers were able to predict ADHD diagnoses with striking accuracy, offering a glimpse into a future where objective, real-time data supports earlier and more personalized mental healthcare.
(2 min. read)

7. Novel pediatric pacemaker shows safety, effectiveness for fragile infants in multi-center study

A novel implantable pacemaker designed specifically for infants has demonstrated safety and effectiveness in stabilizing heart rhythms for at least two years. The multi-center study of 29 infants showed stable pacing, normal electrical parameters and expected battery life, offering a viable alternative to standard-size devices for the smallest children.
(2 min. read)

8. Socioeconomic disadvantage associated with higher long-term mortality for children after heart surgery

Children who had heart surgery and come from less advantaged neighborhoods in the Washington, D.C., region are much more likely to die in the long term than those from neighborhoods with more wealth and opportunity. The finding was part of a presentation titled, Socioeconomic Disadvantage Is Associated with Higher Long-Term Mortality After Cardiac Surgery, by Jennifer Klein, MD, MPH, cardiologist at Children’s National Hospital, during the Society of Thoracic Surgeons Annual Meeting in Los Angeles.
(2 min. read)

9. Children’s National brings AI into the RHD early diagnosis equation

Experts from Children’s National traveled to Uganda to continue work on a pilot program applying artificial intelligence (AI) to the diagnosis of rheumatic heart disease (RHD). The team created a tool that uses AI to predict RHD by identifying leaky heart valves on handheld ultrasound devices, then prompts a referral for a full echocardiogram.
(2 min. read)

10. Fighting food insecurity with fresh produce and education

Food insecurity is rising in Washington, D.C. and it’s hitting families with children the hardest. That’s why Children’s National Hospital created the Family Lifestyle Program (FLiP) – a multi-layered intervention, which offers Patient Navigation (FLiP-PN) and a Produce Prescription Intervention (FLiPRx). FLiP is a Food Is Medicine, clinical-community initiative that helps families get access to fresh food, build healthy habits and lower their risk of diet-related diseases like diabetes and obesity.
(3 min. read)

collage of news outlet logos

Children’s National in the News: 2025

collage of news outlet logosIn 2025, Children’s National Hospital was featured in major national news outlets for pioneering advances in pediatric care, groundbreaking clinical research and powerful human stories of healing and hope. From gene therapy for sickle cell disease and innovative pacemakers for newborns to breakthrough transplants, cancer trials and emerging mental health concerns like AI psychosis, these stories highlight the hospital’s leadership across the full spectrum of pediatric medicine. The following ten highlights showcase the patients, families and experts behind this impact, as reported by outlets including NBC News, The Washington Post, Good Morning America, USA Today, Healio, ABC News and ESPN.

1. Gene therapy treatment gives new hope to sickle cell patients

Children’s National patient Wedam, 19, begins the first steps for intensive gene therapy for sickle cell disease, discussing his skepticism while his mother expresses her joy and gratitude for the treatment. (NBC News)

2. Little pacemakers ‘reliable’ in stabilizing newborns needing pacing

Charles Berul, MD, pediatric electrophysiologist and emeritus chief of Cardiology, discusses his study highlighting the safety and efficacy of an innovative smaller pacemaker designed for newborns with critical congenital heart disease. (Healio)

3. Why this clinical trial is offering some young cancer patients hope

Catherine Bollard, MBChB, MD, senior vice president and chief research officer, and the NexTGen team are poised to recruit patients for a new clinical trial that will take on an old, implacable foe: children’s solid tumors. (The Washington Post)

4. 11-year-old receives living donor heart valve to replace artificial one in breakthrough surgery

Yves d’Udekem, MD, PhD, chief of Cardiac Surgery, talked to Good Morning America about how an 11-year-old’s groundbreaking partial heart transplant will change his life and the lives of other children in need of valve replacements. (Good Morning America)

5. 9-year-old fights brain cancer with magic in her heart

The Lilabean Foundation along with Brian Rood, MD, medical director of the Brain Tumor Institute, talked about how patients like Kasey Zachman are the motivation behind finding a cure for brain cancer. (ABC News)

6. How Little League helps father-son duo recover from kidney transplant

USA Today Sports spoke with Gavin Brown and his parents, as well as Yi Shi, MD, a pediatric nephrologist at Children’s National Hospital, about their kidney transplant journey. (USA Today)

7. Jayden Daniels surprises Children’s National patient

After Jayden Daniels visited Commanders fan Sarah Addison at Children’s National Hospital while she was being treated for myeloid leukemia, they quickly became friends. (ESPN)

8. At D.C. children’s hospital, opera singers offer light, hope and healing

International opera stars performed for children, their families and employees at Children’s National Hospital. (The Washington Post)

9. ‘Gift of life:’ Tiny heart transplant saves life of Maryland baby

A baby boy in Maryland is back home after being given a second chance at life, just before his first birthday. The boy’s mother and his surgeon, Manan Desai, MD, share the remarkable story of a moment that changed all of their lives. (NBC4)

10. AI psychosis: Kids left delusional and paranoid over conversations with chatbots

Ashley Maxie-Moreman, PhD, clinical psychologist, spoke to ABC7 about what AI psychosis is and what parents need to know. (ABC7)

colorful strands of DNA

New insights into how vosoritide affects growth signaling in hypochondroplasia

colorful strands of DNAA new study published in The Journal of Clinical Endocrinology & Metabolism provides insight into how vosoritide, a C-type natriuretic peptide (CNP) analog, affects growth-related biomarkers in children with hypochondroplasia (HCH), revealing a potential interaction between CNP signaling and insulin-like growth factor-1 (IGF-1).

“CNP analogs are novel therapeutics, and their interaction with other growth-promoting peptides has been largely uncharted territory,” said Roopa Shankar, MD, endocrinologist at Children’s National Hospital and lead author of the study.

What the study found

The phase 2 clinical trial followed 24 children with HCH who received daily vosoritide injections for one year. While vosoritide has been shown to promote height gain, little was previously known about its downstream effects on other growth-promoting peptides.

At baseline, children with HCH had elevated levels of amino-terminal propeptide of C-type natriuretic peptide (NTproCNP) and reduced IGF-1 levels. Following treatment with vosoritide, NTproCNP levels declined steadily over 12 months, while increases in IGF-1 were more delayed and variable.

Despite these different response patterns, changes from baseline in the two biomarkers at one year were positively correlated, suggesting a significant, and likely indirect, interaction between growth pathways.

The patient benefit

“These findings are promising and suggest that further study could help us better understand how the different growth signaling pathways interact in other genetic conditions where CNP analogs and growth hormone therapy may both be utilized in concert to improve growth outcomes,” said Dr. Shankar.

Children’s National leads the way

Children’s National is leading clinical trials of vosoritide across multiple genetic conditions, including hypochondroplasia, aggrecan mutations and Rasopathies, under the direction of Andrew Dauber, MD, MMSc, chief of Endocrinology. Additional studies in Turner syndrome are led by Dr. Shankar.

“This work positions us to better understand not only whether these therapies work, but how they work,” the researchers noted, “which is essential for advancing care for children with genetic growth disorders.”

Read the full study, Effect of Vosoritide Therapy on IGF-I and Endogenous C-Type Natriuretic Peptide in Hypochondroplasia, in The Journal of Clinical Endocrinology & Metabolism.

Other Children’s National authors include Andrew Dauber, MD, MMSc, Anqing Zhang, PhD, Niusha Shafaei, MSc, Kimberly Pitner, MSHS, BSN, RN, Raheem Seaforth.

Mother checking child's blood glucose levels

Improving research design in pediatric psychology

Mother checking child's blood glucose levels

The authors use examples from their own trials in pediatric Type 1 and Type 2 diabetes and provide guidance on how to align study design with the research question and hypothesis, consider the stage of the research and field, and consider the importance of ensuring that the treatments developed serve all children.

A new study published in the Journal of Pediatric Psychology offers updated guidance to help pediatric psychology researchers design stronger and more equitable intervention studies, focusing on how to choose the right comparator conditions in clinical trials.

Why it matters

When behavioral researchers want to test a developed intervention, they often conduct a randomized controlled trial. In these studies, the new intervention is compared to something else — a comparator or control condition — with participants randomized to receive one of the two options. This approach helps determine if the intervention being tested helps or makes a difference in what was intended.

Comparator conditions can be designed in many different ways. Researchers aim to balance creating a strong study design — so they can be confident in their findings — with ensuring that treatments are developed and tested in ways that are appropriate and helpful for all children, especially those most affected by a particular illness. Choosing the right comparator is critical for conducting good science and developing effective interventions.

“We are hopeful that our paper will help researchers consider how to design studies to be both rigorous and equitable so that we design and use treatments that are well thought out, well tested and make a difference to the children and families we treat,” said Eleanor Mackey, PhD, psychologist at Children’s National Hospital and senior author of the study.

What’s unique

“We wanted to highlight not just how to choose a comparator condition, but why that choice matters in behavioral research,” said Dr. Mackey.

The authors use examples from their own trials in pediatric Type 1 and Type 2 diabetes and provide guidance on how to align study design with the research question and hypothesis, consider the stage of the research and field, and consider the importance of ensuring that the treatments developed serve all children. They also encourage researchers to seek input from patients, families and other stakeholders early in the design process to ensure studies are both scientifically sound and equitable.

Moving the field forward

“The families we serve have to be able to trust the research we conduct and know that we are providing the best possible options for care,” added Dr. Mackey. “Taking the time to consider how these studies are conducted is an important step in that effort.”

Additional Children’s National author – Randi Streisand, PhD, CDCES.

Read the full study, Rigor and equity in intervention study design in pediatric psychology: a focus on comparator conditions from diabetes research, in the Journal of Pediatric Psychology.

US News badges

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.

Poster presentation at the Pediatric Endocrine Society annual meeting

Encouraging early results for vosoritide in Turner syndrome

Poster presentation at the Pediatric Endocrine Society annual meeting

Roopa Shankar, MD, an endocrinologist at Children’s National Hospital and principal investigator on the study, presented these findings at the Pediatric Endocrine Society annual meeting.

In the world’s first clinical trial of vosoritide for Turner Syndrome (TS), preliminary results show an increase in annualized growth velocity (AGV) in girls with TS in the first six-months of the trial.

Roopa Shankar, MD, an endocrinologist at Children’s National Hospital and principal investigator on the study, presented these findings at the Pediatric Endocrine Society annual meeting on May 17.

The big picture

TS is a rare genetic disorder that occurs in one to about 2,500 girls and is caused by a partial or complete missing X chromosome. Some of the characteristics of TS are short stature, delayed puberty, kidney, thyroid and heart problems. Although there is no cure for TS, many of the symptoms can be treated.

Previously approved for use in achondroplasia, vosoritide is a C-type natriuretic peptide (CNP) analog that binds its receptor on healthy cartilage cells called chondrocytes, leading to increased chondrocyte proliferation and differentiation via its inhibition of the ERK1/2-MAPK pathway.

To date, four participants are enrolled in the trial, and three have completed six months of treatment. Participants with prior growth hormone (GH) exposure increased AGV by +4.9 and +1.57 cm/year over the baseline AGV on GH treatment. The participant without prior GH exposure had an increased AGV +6.04 cm above the baseline at the six-month visit.

What’s next

“The increase in AGV with vosoritide is comparable to the response expected with GH treatment in the GH naïve patient,” says Dr. Shankar. “What’s particularly encouraging is the higher AGV seen in patients previously treated with GH — compared to their baseline on GH alone—which suggests vosoritide may offer additional growth benefits, even when GH responsiveness has diminished. While these preliminary results are very promising, we need longer-term data to establish the safety and the sustained impact on growth. Recruitment for this study is ongoing.”

Find out more about the trial here.

Additional authors from Children’s National include: Niusha Shafaei, MSc, Anqing Zhang, PhD, Kimberly Pitner, MSHS, BSN, RN, Niti Dham, MD, Andrew Dauber, MD, MMSc.

Poster session at REI Week 2025

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.
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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.

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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.

Cast your vote
Andrew Dauber, MD, MMSc, at work

Breaking barriers in growth disorder treatment for families

Andrew Dauber, MD, MMSc, at work

Children with hypochondroplasia were previously left with no options for effective long-term treatment, but Andrew Dauber, MD, MMSc, and his team continue to see promising results for treating children with hypochondroplasia using vosoritide.

Paving new roads for families at dead ends

For many children with short stature and other rare genetic growth disorders, there have been no next steps after usual treatment options prove ineffective. Researchers at Children’s National Hospital are digging deeper to find the root genetic causes of short stature disorders and creating novel, nuanced treatment options that have the opportunity to change how the field approaches these cases. From the creation of the growth specialty clinic to creating a study for one patient, the endocrinology team at Children’s National is focused on treating children with the uniqueness that their growth disorders require.

Hypochondroplasia

Many short stature disorders are caused by genetic variations that slow the growth of cells in the growth plate.

Children with hypochondroplasia were previously left with no options for effective long-term treatment, but Andrew Dauber, MD, MMSc, chief of Endocrinology at Children’s National, and his team continue to see promising results for treating children with hypochondroplasia using vosoritide. This drug had previously been approved for treatment of children with achondroplasia, the most common form of dwarfism. Treatment of hypochondroplasia has shown similar and, often, even better results. Study participants have managed the treatment well and have overall been very satisfied with the results.

Children’s National is the only site in the world offering this treatment to patients. The study includes over 50 children from two distinct subsets of patients — those with hypochondroplasia and those with other genetic short stature disorders such as RASopathy conditions, most common of which is Noonan syndrome, and children with mutations in the aggrecan and NPR2 genes. The study has found a significant increase in growth rates for children with hypochondroplasia who underwent treatment with vosoritide for one year.

“These are the first patients in the world to ever receive this medication for their conditions,” said Dr. Dauber. “The results are very promising and may change the way we practice medicine. Patients have come from all over the world to participate in the study.”

The preliminary data is even more promising for children in the study with other genetic conditions, which include defects that are more directly related to C-natriuretic peptide (CNP), which vosoritide targets directly. This is the first medication that directly targets the pathway in chondrocytes (cells in the growth plate that make the bones grow longer) affected by these specific mutations. Those patients are still undergoing their first full year of treatment and results of that section of the study are expected to be released next fall.

“We’re really starting to see this therapeutic landscape open up and develop for patients with this rare condition, for which right now there is no approved therapy, so really exciting times in this space,” says Dr. Dauber.

ACAN study

Dr. Dauber was a part of a study that was the first-of-its-kind to provide genetic testing for children with short stature and their families, finding ACAN gene mutations in multiple family members, and providing hormone therapy to the children impacted. The ability to diagnose this type of gene mutation allows families to be proactive with treatments — both as their child is growing and how other family members can reduce the risk of further complications down the road.

“We’re at the tip of the iceberg with research that explores this gene mutation,” says Dr. Dauber.

Study of one

This type of unique and cutting-edge research isn’t new to the endocrinology team at Children’s National, who is focused on creating unique interventions to find answers for patients and families. When a patient with short stature was found to have a unique mutation in his growth hormone receptor, Dr. Dauber and his team created a single patient trial with a precision medicine approach to overcome the patient’s growth hormone resistance. This isn’t the first time Dr. Dauber led a single patient study. Even when study populations are small, unlocking genetic answers and treatment options for even one patient is at the core of work being done at Children’s National. Dr. Dauber emphasized the importance of these findings for the medical community, particularly for those dedicated to pediatric endocrinology. He noted that understanding the nuanced responses among different children is crucial for optimizing future treatments.

What’s next

Even with improved height and growth outcomes, there is still more to uncover. In addition to the world’s first clinical trial using vosoritide in children for hypochondroplasia, Children’s National researchers are studying the quality of life for children with the disorder and how it may be affected by treatment, aiming to provide this full scope of care for children with these rare conditions. Dr. Dauber and his team continue to study connections between genetic biomarkers and response rates to clinical therapies, with hopes of discovering how these targeted approaches to treatment can be most effective. The work done by the team at Children’s National has shown results to warrant phase three of the trial.

“This ongoing commitment to innovative research underscores the relentless pursuit of targeted therapies, bridging gaps and bringing hope to families and patients worldwide,” says Dr. Dauber.

Dr. Dauber and patient

Innovative treatment for growth disorders

Dr. Dauber and patient

Andrew Dauber, M.D., M.M.Sc., chief of Endocrinology, with a patient.

Andrew Dauber, M.D., M.M.Sc., chief of Endocrinology, leads a program that brings together comprehensive resources for children with rare genetic growth disorders, including basic science, translational and clinical research.

“Discovery is important, and research gives us many answers,” he says. “But what we do with those answers is what really matters. There’s nothing better than seeing the impact our research is having on an individual patient. It is so gratifying to hear from parents about how their kids are making progress thanks to a protocol we developed.”

Mia thrives with the right approach

Middle schooler Mia loves to dance, practice gymnastics and hang out with friends. She was born in Croatia with hypochondroplasia, a genetic disorder that slows cell growth and causes short stature and limb shortening.

Mia practices her dance moves.

Mia practices her dance moves.

She struggled in kindergarten because she was so small. “Kids can be very mean if you’re different,” says Mia’s dad, Ivan. “For years, we took Mia to different specialists in Europe without getting the help she needed. I researched endocrinologists all over the world. They all pointed us to the United States.” This led Ivan to Children’s National and Dr. Dauber.

Dr. Dauber invited the family to the U.S. to participate in a clinical trial that was to start very soon. “I knew this was our only chance,” said Ivan, who rushed to bring his family to Washington, D.C., for the initial screening appointments. They later returned for more measurements, Mia’s first dose of medicine and a three-month supply to go. The family returns to Children’s National every six months.

In Mia’s first year, she grew more than 3 inches. Her arms also grew longer. “Dr. Dauber is probably the best doctor in the world,” Ivan says. “He is like a friend to Mia and has helped us make sure she has as normal a life as possible. This growth, including in her confidence, has been life-changing for her.” The family relocated to the U.S., and Mia will continue in the trial until she reaches puberty.

“Now, the difference between Mia and other kids is much less,” Ivan says. “She makes new friends more easily and is a happy, happy kid. As for me, I want to cry for how happy I am. Dr. Dauber and Children’s National made it all possible.”

collage of news logos

Children’s National in the News: 2024

collage of news logosIn 2024, Children’s National Hospital continued to make remarkable strides across diverse areas of pediatric medicine, from groundbreaking technological innovations to critical health advocacy. The following compilation showcases ten significant stories that demonstrate the breadth and depth of the hospital’s impact, as featured in major national news outlets including NBC Nightly News, CNN, The Washington Post, The New York Times, NPR, The Today Show, Healio, and POLITICO. Delve into our 2024 news highlights for more.

1. World’s smallest pacemaker gives new hope to babies with heart defects

Charles Berul, M.D., and a patient family talk about the pill-sized pacemaker that saved the life of Abby, an infant born with deadly heart defects. (NBC Nightly News)

2. ‘A $10 death trip’: Fentanyl is killing teens. Meet one fighting for his life

Sivabalaji Kaliamurthy, M.D., addiction psychiatrist and director of the Addictions Program, spoke to CNN about the impact of drug addiction on teen health and the lack of resources available to treat opioid use disorder. (CNN)

3. Health panel urges interventions for children and teens with high BMI

Susma Vaidya, M.D., M.P.H., associate medical director of the IDEAL Clinic, shared her concerns about childhood obesity treatment recommendations issued today by a leading panel of independent U.S. health experts. (The Washington Post)

4. An Rx for food? Doctor’s offices offer groceries to those in need

Shideh Majidi, M.D., M.S.C.S., and Emily Frymark, clinical dietitian, spoke about how the food pharmacy, created in partnership with the Capital Area Food Bank, benefits patients with diabetes and other chronic conditions. (The Washington Post)

5. First patient begins newly approved sickle cell gene therapy

Kendric Cromer, a 12-year-old boy being treated at Children’s National Hospital, became the first person in the world with sickle cell disease to begin a commercially approved gene therapy that may cure the condition. “This is a big effort,” says David Jacobsohn, M.D., ScM, M.B.A. (The New York Times)

6. ‘We created this problem’: A pediatric surgeon on how gun violence affects children

Mikael Petrosyan, M.D., associate chief of General and Thoracic Surgery, discusses the stress medical staff face when treating young victims of gun violence. (NPR)

7. 7th grade boy rings bell after final round of chemotherapy

Landon, an 11-year-old patient, rang the bell at Children’s National Hospital with family, friends, doctors and nurses cheering after finishing his final round of chemotherapy. (The Today Show)

8. Study: One in three adolescents experience ‘period poverty’

Monika Goyal, M.D., M.S.C.E., pediatric emergency medicine specialist and co-director of the Center for Translational Research, emphasized the need for awareness in addressing period poverty in teenagers and young adults. (Healio)

9. The AI assurance labs are coming

Kolaleh Eskandanian, Ph.D., M.B.A., P.M.P., vice president and chief innovation officer, participates in a panel discussion covering AI data collection, associated risks, reliance and other topics related to artificial intelligence. (POLITICO)

10. First day of a ‘new life’ for a boy with sickle cell

Children’s National patient Kendric Cromer, 12, became one of the first children ever to be treated with a newly approved gene therapy that will free him from the sickle cell disease that has stolen his childhood. (The New York Times)

the 8th Annual 2024 T1DX-QI logo

Children’s National shares insights at 2024 T1DX-QI Learning Session

the 8th Annual 2024 T1DX-QI logoExperts from Children’s National Hospital presented research at the 8th Annual 2024 T1DX-QI (Type 1 Diabetes Exchange Quality Improvement Collaborative) Learning Session held in Chicago, Ill. This session emphasizes delivering equitable, evidence-based and cost-effective diabetes care through integrating clinical science, quality improvement, technology and advocacy.

Oral Presentations:

  • Assessing Readiness to Transition to Adult Care among Young Adults with T1D by Jody Grundman, M.D.
  • Improving Microalbuminuria Screening Rates Among Pediatric Diabetes Patients: A Clinic-Wide Initiative by Sarah Lydia Holly, BSN, RN.
  • Addressing Disparities in Diabetes Care: Implementing SDOH Screening at Diagnosis by Sarah Lydia Holly, BSN, RN.
  • Developing a Tracking Tool for Insulin Pump Prescriptions Among Children and Adolescents with Type 1 & Type 2 Diabetes by Amanda Perkins, N.P., C.P.N.P., M.P.H.

Learn more about these presentations here.

2024 with a lightbulb instead of a zero

The best of 2024 from Innovation District

2024 with a lightbulb instead of a zero2024 marked another groundbreaking year for Children’s National Hospital, showcasing remarkable advances across the spectrum of pediatric medicine, research and healthcare innovation. From pioneering surgical procedures to breakthrough artificial intelligence applications, the institution continued to push the boundaries of what’s possible in children’s healthcare. Read on for our list of the most popular articles we published on Innovation District in 2024.

1. Prenatal COVID exposure associated with changes in newborn brain

A study led by researchers at Children’s National Hospital showed that babies born during the COVID-19 pandemic have differences in the size of certain structures in the brain, compared to infants born before the pandemic. The findings suggest that exposure to the coronavirus and being pregnant during the pandemic could play a role in shaping infant brain development.
(3 min. read)

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

Children’s National Hospital was ranked as a top hospital in the nation by the U.S. News & World Report 2024-25 Best Children’s Hospitals annual rankings. This marks the eighth 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.
(2 min. read)

3. Children’s National performs first ever HIFU procedure on patient with cerebral palsy

In January 2023, a team of multidisciplinary doctors performed the first case in the world of using bilateral high intensity focused ultrasound (HIFU) pallidotomy on Jesus, a 22-year-old patient with dyskinetic cerebral palsy. The procedure is part of a clinical trial led by Chima Oluigbo, M.D., pediatric neurosurgeon at Children’s National Hospital.
(3 min. read)

4. Novel ultrasound device gets FDA breakthrough designation with Children’s National support

A novel ultrasound device developed by Bloom Standard received the Food and Drug Administration’s valued breakthrough device designation with the help of Children’s National Hospital. The device that enables autonomous, hands-free ultrasound scans to be performed anywhere, by any user.
(2 min. read)

5. First-of-its-kind pilot study on the impacts of Lyme disease in pregnancy and infant development

Understanding the effects of Lyme disease on the developing fetal brain is essential to ensure timely prenatal and postnatal treatments to protect the fetus and newborn. In response to this need, Children’s National Hospital is leading a pilot study to establish the groundwork needed for a larger study to determine the effect of in utero exposure to Lyme disease on pregnancy and early childhood neurodevelopmental outcomes.
(3 min. read)

6. Earliest hybrid HLHS heart surgery kids thrive 5 years later

Five years ago, Cayden was born 6 weeks early weighing less than four pounds and at risk of dying from her critical congenital heart disease. Today, she’s a happy five-year-old. Early diagnosis of her hypoplastic right ventricle, double inlet left ventricle and critical coarctation of the aorta allowed for the team at Children’s National Hospital to create a careful plan for safe delivery and to offer an innovative hybrid HLHS surgical approach at the hospital within 24 hours after she was born.
(1 min. read)

7. Wayne J. Franklin, M.D., F.A.C.C., named senior vice president of the Children’s National Heart Center

Children’s National Hospital appointed Wayne J. Franklin, M.D., F.A.C.C., as the new senior vice president (SVP) of the Children’s National Heart Center. In this role, Dr. Franklin oversees the full spectrum of heart care services including cardiac imaging and diagnostics, interventional cardiology, electrophysiology, cardiac anesthesia, cardiac surgery and cardiac intensive care.
(2 min. read)

8. Artificial – and accelerated – intelligence: endless applications to expand health equity

By pioneering artificial intelligence (AI) innovation programs at Children’s National Hospital, Marius George Linguraru, D.Phil., M.A., M.Sc., and the AI experts he leads are ensuring patients and families benefit from a coming wave of technological advances. The team is teaching AI to interpret complex data that could otherwise overwhelm clinicians.
(4 min. read)

9. Evidence review: Maternal mental conditions drive climbing death rate in U.S.

Painting a sobering picture, a research team led by Children’s National Hospital culled years of data demonstrating that maternal mental illness is an under-recognized contributor to the death of new mothers. They called for urgent action to address this public health crisis.
(3 min. read)

10. Nathan Kuppermann, M.D., M.P.H., named chief academic officer and chair of Pediatrics

Children’s National Hospital appointed Nathan Kuppermann, M.D., M.P.H., as its new executive vice president, chief academic officer and chair of Pediatrics. In this role, Dr. Kuppermann oversees research, education and innovation for the Children’s National Research Institute as well as academic and administrative leadership in the Department of Pediatrics at George Washington University School of Medicine & Health Services.
(2 min. read)

11. First global clinical trial achieves promising results for hypochondroplasia

Researchers from Children’s National Hospital presented findings from the first clinical trial of the medication vosoritide for children with hypochondroplasia – a rare genetic growth disorder. During the phase 2 trial, researchers found vosoritide increased the growth rate in children with hypochondroplasia, allowing them to grow on average an extra 1.8 cm per year.
(2 min. read)

12. Pioneering research center aims to revolutionize prenatal and neonatal health

Since its establishment in July 2023, the Center for Prenatal, Neonatal & Maternal Health Research at Children’s National Hospital has gained recognition through high-impact scientific publications, featuring noteworthy studies exploring the early phases of human development.
(3 min. read)

ISPAD logo

Children’s National researchers joined ISPAD’s 50th anniversary conference

ISPAD logoResearchers from Children’s National Hospital participated in the 50th anniversary conference of the International Society for Pediatric and Adolescent Diabetes (ISPAD) in Lisbon, Portugal. As the only global organization dedicated to all forms of childhood diabetes, ISPAD promotes clinical and basic science, research, education and advocacy for children and adolescents with diabetes. The conference provided members worldwide with a platform to exchange scientific and clinical insights on pediatric and adolescent diabetes.

Poster presentations:

The participation of Children’s National researchers highlights their commitment to advancing diabetes care and research for children and adolescents. By collaborating with global experts, they continue to contribute collective efforts to improve outcomes for young people living with diabetes worldwide.

Learn more about these presentations here.

test tubes filled with blood and serum

Pharmacokinetic and pharmacodynamic analysis of vosoritide in Phase 2 trial

test tubes filled with blood and serum

The research, published in Hormone Research in Paediatrics, examined whether pharmacokinetic (PK) factors or other blood biomarkers correlated with the response to treatment of vosoritide in children with hypochondroplasia.

Researchers from Children’s National Hospital presented new data from the world’s first clinical trial of vosoritide use for growth disorders at the European Society for Pediatric Endocrinology annual meeting. The research, published in Hormone Research in Paediatrics, examined whether pharmacokinetic (PK) factors or other blood biomarkers correlated with the response to treatment of vosoritide in children with hypochondroplasia. The study found that drug exposure ­­— as measured by average PK area under the curve (AUC) — did not correlate with any growth outcome.

The big picture

Previously published results of the clinical trial of vosoritide in children with hypochondroplasia showed that vosoritide can help kids with hypochondroplasia grow better. For this study, the researchers analyzed PK parameters by non-compartmental methods. Pharmacodynamic markers were measured at day 1, the 6-month and 12-month visits. The PK analysis indicates that drug exposure was correlated to global C-type natriuretic peptide (CNP) activity as measured by urine cyclic guanosine monophosphate (cGMP) concentrations.

Moving the field forward

There are currently no approved therapies for hypochondroplasia. Children’s National is the first site in the world to conduct a trial of a targeted treatment for hypochondroplasia and this is the first study to report PKs of vosoritide in children with hypochondroplasia.

“The results of our study showed that the drug levels achieved are similar to what was achieved in patients with achondroplasia,” says Andrew Dauber, M.D., M.M.Sc., chief of Endocrinology at Children’s National and one of the study authors. “This suggests that the dose we used is a reasonable one, paving the way for a Phase 3 trial of vosoritide in children with hypochondroplasia.”

What’s next

Dr. Dauber emphasized the importance of these findings for the medical community, particularly for those dedicated to pediatric endocrinology. He noted that understanding the nuanced responses among different children is crucial for optimizing future treatments.

“This ongoing commitment to innovative research underscores the relentless pursuit of targeted therapies, bridging gaps and bringing hope to families and patients worldwide,” says Dr. Dauber.

Read the study in Hormone Research in Paediatrics: Phase 2 Trial of Vosoritide Use in patients with Hypochondroplasia: Pharmacokinetic/ Pharmacodynamic analysis from 12 Month Data

photograph of a pile of insulin pumps

CME Webinar: Delaying Type 1 Diabetes: Early Antibody Screening and Use of Teplizumab in Children

In this webinar, Shideh Majidi, M.D., M.S.C.S., director of the Childhood and Adolescent Diabetes Program at Children’s National Hospital, discussed early antibody screening and use of teplizumab in children.

Key takeaways

  • Current state of type 1 diabetes: Type 1 Diabetes (T1D) is characterized by absolute insulin deficiency due to pancreatic beta-cell destruction, often diagnosed in childhood but can occur in adults as well. It requires lifelong insulin management.
  • Increased risk and screening: Individuals with family members who have T1D are at higher risk of developing T1D, but most newly diagnosed cases have no family history. Early screening can help reduce diabetic ketoacidosis (DKA) rates and improve management.
  • Stages of type 1 diabetes: T1D is classified into three stages: Stage 1 (normal blood sugars with 2 or more autoantibodies), Stage 2 (dysglycemia with 2 or more autoantibodies), and Stage 3 (clinical T1D requiring insulin).
  • Early screening and follow-up: Screening for diabetes autoantibodies can be done through various programs and labs. Follow-up involves regular monitoring based on the number of antibodies and blood sugar levels. If patients have antibodies or abnormal blood sugars, they can be referred to the T1Delay Program at Children’s National (through an endocrine/diabetes referral).
  • Teplizumab (T-zield) treatment: T-zield is an FDA-approved treatment for delaying the onset of Stage 3 T1D in individuals aged 8 and older with Stage 2 T1D. It involves a 14-day infusion regimen and has been shown to delay insulin need by a median of 2 years.
  • Clinical implementation and support: Children’s National offers T-zield treatment through a multi-departmental effort. The program aims to improve both quality of life and clinical outcomes for patients.

More information

  • For more information on this webinar, as well as access to the presentation slide deck, visit our website.
  • To register for future CME opportunities or view past presentations, visit childrensnational.org/Webinars.
2024-25 US News Badges

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

2024-25 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 2024-25 Best Children’s Hospitals annual rankings. This marks the eighth 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.

This year, U.S. News ended ordinal rankings on its Honor Roll. Instead of assigning a numerical rank from 1 to 10, all hospitals on the Honor Roll will be recognized as having attained the highest standards of care in the nation.

In addition, Children’s National tied for #1 pediatric hospital in the Mid-Atlantic region, which includes New York, New Jersey, Delaware, Pennsylvania, the District of Columbia, West Virginia and Virginia. It’s also best in the Mid-Atlantic in Neonatology.

For the fourteenth straight year, Children’s National ranked in 10 specialty services. New this year, U.S. News included behavioral health as a service line in the rankings. Since it’s the first year, there are no ordinal rankings for behavioral health, but the Children’s National program was named one of the top 50 programs in the country.

“In my first year here, I witnessed what makes Children’s National so special — our commitment to collaboration, empowering one another, and charting a bold path forward for pediatric care,” said Michelle Riley-Brown, MHA, FACHE, president and chief executive officer of Children’s National. “I’m proud U.S. News again recognized Children’s National as one of the top in the nation and the highest-ranked pediatric hospital in D.C., Maryland and Virginia. Together, we’ll continue to push the boundaries of care, research and innovation to make a difference for those who matter most — the kids.”

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.

“For nearly two decades, U.S. News has published Best Children’s Hospitals to empower the parents and caregivers of children with complex medical needs,” said Ben Harder, chief of health analysis and managing editor at U.S. News. “Children’s hospitals appearing on the U.S. News Honor Roll have a track record of delivering unparalleled specialized care.”

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 four specialties ranked among the top 50 are Behavioral Health, Cardiology and Heart Surgery, Pulmonology and Lung Surgery, and Urology.

teens hiking in the woods

Mindfulness-based interventions boost teens with Type 1 diabetes

teens hiking in the woodsA team of experts at Children’s National Hospital, using the input of teens with Type 1 diabetes from our Children’s National clinics, adapted a six-week virtual group diabetes education program and mindfulness-based intervention. In their new study published in Journal of Pediatric Psychology, the team described how the intervention might work to support teens with Type 1 diabetes experiencing anxiety or depression.

The findings showed that both the mindfulness-based intervention and the diabetes education program were successfully implemented and valued by the participating teens. While the study was limited in scope, focusing primarily on assessing the programs’ acceptance and perceived value among adolescents, preliminary results suggest participation in the programs may also contribute to improvements in mood, diabetes-related distress and glycemic control.

Eleanor Mackey, Ph.D., psychologist and author of the study, shared more about the learnings.

Q: What’s been the hold-up in the field?

A: Many adolescents with Type 1 diabetes experience symptoms of anxiety and depression, yet it can be hard to access care to relieve some of these symptoms. Group-based, brief virtual care may be a way to provide more access to necessary support. Testing whether such programs are desired and useful by teens and their families, and whether they show promise for improving symptoms of depression and anxiety, is an important step in addressing this gap in care.

Q: How does this work move the field forward?

A: This study helped us to use the input of the teens we hope to serve to create and adapt programs that they were enthusiastic about and test them to see if they were helpful. We used these findings to obtain a second grant from the National Institutes of Health to do a larger scale study to see whether these programs help with depression, anxiety, eating, stress and diabetes management.

Q: What’s the goal and how will this work benefit patients?

A: Our ultimate goal is to create easy to access programs which help improve anxiety, depression and diabetes management in teens with Type 1 diabetes. We are trying to ensure that these programs are feasible and accessible and could be translated into our clinics if they prove to be beneficial. We hope to improve the ways we identify teens experiencing anxiety and depression and provide them the option for this brief group-based care in our clinics.

Q: What did you find that excites you and what’s next?

A: We were very excited to find that teens were interested in enrolling in these programs and found them useful. We were also excited to see that our very preliminary data indicates that mindfulness-based interventions may improve depression and even possibly glycemic control.

Children’s National is the first institution that we know of to use teen feedback to adapt a mindfulness-based intervention specific to teens with Type 1 diabetes. Our team is now preparing to embark on a five-year project in collaboration with the Barbara Davis Center in Colorado to evaluate the effectiveness of these programs with a larger group of teens.

Additional authors from Children’s National include: Katherine Patterson Kelly, Ph.D., R.N.; Randi Streisand, Professor, Ph.D., CDCES; and Jack Vagadori.

American Diabetes Association 84th Scientific Sessions logo

Children’s National at ADA 84th Scientific Sessions

American Diabetes Association 84th Scientific Sessions logoThe American Diabetes Association 84th Scientific Sessions were held in Orlando, Florida. The meeting presented an opportunity for researchers and health care professionals to exchange knowledge on the latest scientific advances and breakthroughs in diabetes. Participants included:

Oral Presentations:

Poster Presentations: