During the meeting, Andrew Dauber, MD, MMSc, chief of Endocrinology and director of the Center for Precision Medicine and Genomics Research, was formally presented with the ESPE International Research Award.
Endocrinologists from Children’s National attended the 64th annual European Society for Paediatric Endocrinology (ESPE) meeting this September in Marseille, France. Each year, leading pediatric endocrinology and diabetes experts from around the world gather to share the latest research, clinical advances and innovations shaping the field.
During the meeting, Andrew Dauber, MD, MMSc, chief of Endocrinology and director of the Center for Precision Medicine and Genomics Research, was formally presented with the ESPE International Research Award. The award recognizes individuals whose research has made an exceptional and lasting impact on basic endocrine science or clinical pediatric endocrinology.
Dr. Dauber, a pediatric endocrinologist who specializes in growth disorders, has published more than 150 studies and is internationally recognized for his work identifying rare genetic growth conditions, uncovering genetic factors that influence height and puberty, and advancing precision medicine approaches for children with endocrine disorders.
A strong advocate for integrating research and clinical care, Dr. Dauber leads efforts to translate genomic discoveries into new treatments and improved patient care. His most recent work includes a Phase 3 multicenter study published in NEJM Evidence that found vosoritide increases growth in children with hypochondroplasia, a rare genetic skeletal condition with limited treatment options.
In addition to the award presentation, Dr. Dauber and Roopa Kanakatti Shankar, MD, shared the latest Children’s National research and clinical insights at the meeting.
https://innovationdistrict.childrensnational.org/wp-content/uploads/2026/09/Dauber-ESPE-2026-feature.jpg300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2025/09/InnovationDistrict_CN_WebHeader-1396px-1030x151.pngInnovation District2026-09-18 15:35:172026-09-18 15:38:45Highlights from the 2026 European Society for Paediatric Endocrinology meeting
Children’s National in Washington, D.C., was once again ranked as a top hospital in the nation by the U.S. News & World Report 2026-27 Best Children’s Hospitals annual rankings. This marks the 10th straight year Children’s National has made the Honor Roll, a distinction awarded to only 10 children’s hospitals nationwide.
Children’s National also ranked in all 11 specialty services and is the highest U.S. News ranked children’s hospital in Washington, D.C, Maryland and Virginia. Recently, 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 third year in a row.
“We’re so proud to reach this milestone of being named among the nation’s top children’s hospitals for the 10th year in a row,” said Michelle Riley-Brown, MHA, FACHE, president and chief executive officer of Children’s National. “Our leadership in specialties like neurology, cancer, neonatology, and diabetes and endocrinology underscores our belief that kids are our everything. It also shows what’s possible when our teams bring their expertise, commitment and heart to make sure every child receives the best care.”
The annual rankings are the most comprehensive source of quality-related information on U.S. pediatric hospitals and recognize the nation’s top 50 pediatric hospitals based on a scoring system developed by U.S. News.
“Earning a place on U.S. News’ Best Children’s Hospitals list is a testament to Children’s National’s dedication to clinical excellence and patient safety,” said Ben Harder, chief of health analysis and managing editor at U.S. News. “These rankings highlight the standout hospitals in each specialty and each region.”
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 specialties ranked in the top 10 nationally by U.S. News are:
https://innovationdistrict.childrensnational.org/wp-content/uploads/2026/09/USNWR-Badges-2026-27-CNRI-1.jpg385685Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2025/09/InnovationDistrict_CN_WebHeader-1396px-1030x151.pngInnovation District2026-09-15 01:00:552026-09-11 14:02:41Children’s National ranked among top children’s hospitals nationwide
Dr. Dauber presented the findings at the 64th Annual Meeting of the European Society for Paediatric Endocrinology (ESPE).
A Phase 3 multicenter study, published in NEJM Evidence and presented at the 64th Annual Meeting of the European Society for Paediatric Endocrinology (ESPE), found that vosoritide may increase growth in children with hypochondroplasia, a rare genetic skeletal condition with limited treatment options.
After 52 weeks, children treated daily with vosoritide had a 2.33 cm per year greater improvement in annualized growth velocity than those who received a placebo. Researchers also reported significant improvements in standing height, height Z-score and arm span, with no new safety signals.
“Hypochondroplasia can affect a child’s growth, physical function, and everyday life, with families often navigating unique challenges as they support their child’s development,” said Dr. Dauber. “The improvements we observed in annualized growth velocity and arm span provide encouraging evidence of how children with hypochondroplasia responded to treatment throughout the study. These findings deepen our understanding of the condition while reinforcing VOXZOGO’s potential as the first targeted medicine developed specifically for children with hypochondroplasia.”
Dr. Dauber delivered the oral presentation at the 2026 meeting in Marseille, France. Participants will continue to be followed in a long-term extension study.
Leading the way
Researchers at Children’s National led the first ever trial of vosoritide for short stature not caused by achondroplasia. The Phase 2 basket trial found increased growth in children with hypochondroplasia and three other rare genetic growth disorders – RASopathies, ACAN mutations and NPR2 deficiency, while the world’s first vosoritide trial for Turner syndrome reported encouraging early growth results.
https://innovationdistrict.childrensnational.org/wp-content/uploads/2026/09/Dauber-presentation-feature-1.jpg300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2025/09/InnovationDistrict_CN_WebHeader-1396px-1030x151.pngInnovation District2026-09-09 10:54:462026-09-09 10:56:15Phase 3 study shows vosoritide improves growth in children with hypochondroplasia
The recommendations cover growth patterns, x-rays, FGFR3 testing, family history and prenatal findings.
Diagnosing hypochondroplasia can be challenging. The signs can be subtle, especially in young children, and the condition can look different from one person to another. Now, an international group of clinicians, researchers and patient representatives has developed the first consensus recommendations to make diagnosis clearer and more consistent.
Hypochondroplasia is a rare genetic skeletal condition associated with disease-causing variants in the FGFR3 gene. It often causes disproportionate short stature and relative microcephaly, or a head size that is larger in proportion to the body.
But there’s no single way that the condition looks. Signs may be difficult to spot in infancy, and children with milder or atypical features can be especially challenging to diagnose. Getting the diagnosis right matters. Without clear answers, families may face delays in getting the monitoring and care their child needs.
The hold-up in the field
Until now, clinicians around the world haven’t had a shared set of criteria for diagnosing hypochondroplasia. To address the gap, international experts and patient representatives reviewed the available evidence and drew on their collective experience. Using a two-stage Delphi process, they reached consensus on a standardized approach to diagnosis.
The new framework includes major and minor diagnostic criteria and categories for definitive, suspected and genetically unconfirmed hypochondroplasia. Clinicians start with a patient’s physical and clinical features, using genetic testing to confirm or clarify the diagnosis when available.
Moving the field forward
The recommendations cover growth patterns, x-rays, FGFR3 testing, family history and prenatal findings. They also offer guidance on when brain magnetic resonance imaging (MRI) may be helpful, including for children with seizures, developmental concerns or increasing head circumference.
“There have been different practices around the world for evaluating children for this diagnosis, and it was gratifying to have all of us agree on a unified approach,” Dr. Dauber said. “These recommendations give clinicians a clear reference for evaluating patients and reaching a definitive diagnosis.”
The framework is designed to work across healthcare settings, including where genetic testing isn’t readily available.
The patient benefit
For families, a more consistent approach could mean getting answers sooner and reducing uncertainty. An earlier diagnosis can also help connect children with appropriate monitoring, developmental services, genetic counseling and other care.
That consistency is becoming more important as potential growth-modifying treatments advance through clinical trials. The new guidelines do not determine who is eligible for treatment, but they can help clinicians and researchers identify patients using the same standards.
Children’s National is a leading center for hypochondroplasia care and research and serves as the lead site for several clinical trials. Next, the team plans to evaluate how well the recommendations work in everyday clinical practice and refine them as new evidence emerges.
Children’s National researchers and clinicians brought a broad portfolio of pediatric diabetes research to the American Diabetes Association 2026 Scientific Sessions in New Orleans, Louisiana, highlighting how multidisciplinary innovation is shaping diabetes care for children and adolescents. Through oral and poster presentations, the team shared work focused on early-stage Type 1 diabetes screening, youth-onset Type 2 diabetes, behavioral health, nutrition and diabetes technology – reflecting a comprehensive approach to improving outcomes for patients and families.
Oral presentations
The oral presentations highlighted key questions in pediatric diabetes care, from early-stage Type 1 diabetes screening and psychosocial support to treatment strategies and nutrition-focused interventions for youth with Type 2 diabetes.
The researchers showcased the scope of pediatric diabetes research in their poster presentations, spanning cardiometabolic risk, diabetes technology, behavioral health and family-centered care.
Together, the presentations reflected the breadth of work by researchers in pediatric diabetes – from early detection and psychosocial support to nutrition, technology and long-term risk reduction. Just as important, they illustrated how collaborative research can translate into more responsive, family-centered care for children living with diabetes and related metabolic conditions.
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The study demonstrates that CNP analogs have the potential to promote growth in children affected by these rare genetic disorders, opening doors to new treatment options.
In the first ever trial of vosoritide for short stature not caused by achondroplasia, researchers found an increase in growth for children with three rare genetic growth disorders where the RAS-MAPK pathway is affected – RASopathies, ACAN and NPR2 deficiency. Vosoritide, an analog of C-type natriuretic peptide (CNP), had previously been approved for treatment of children with achondroplasia.
The study, published in The Journal of Clinical Endocrinology & Metabolism, also reported that children with hypochondroplasia experienced increased growth, a finding previously presented by researchers at Children’s National Hospital.
The big picture
This phase 2 basket trial evaluated 30 participants between the ages of 3 and 11 years old with rare, genetic causes of short stature, all meeting a height requirement of less than -2.25 standard deviation score (SDS). The trial included a 6-month observation period followed by 12 months of vosoritide treatment. During the observation period, participants grew at an average rate of 4.53 cm per year. Once they began the therapy, their growth rate increased to an average of 8.09 cm per year – an increase of 3.56 cm per year.
The study demonstrates that CNP analogs have the potential to promote growth in children affected by these rare genetic disorders, opening doors to new treatment options. However, the authors emphasize the need for careful monitoring during long-term therapy, particularly for patients with aggrecan deficiency, due to the risk of developing conditions such as genu valgum and slipped capital femoral epiphyses.
The hold-up in the field
“Many patients do not get a comprehensive evaluation, including genetic testing, to identify the underlying cause of their short stature,” said Andrew Dauber, MD, MMSc, chief of Endocrinology at Children’s National and lead author of the trial. “In the last decade, our understanding of the genetics of growth disorders has made huge advances, and we can now identify new genetic subgroups of short stature.”
Dr. Dauber led an international team of experts in publishing new consensus guidelines on the genetic evaluation of short stature. Until recently, the only option for treatment has been growth hormone.
“Patients with NPR2 mutations had a particularly robust response to treatment, and so far, have not suffered any significant adverse events,” said Dr. Dauber. “Because NPR2 is the gene responsible for the CNP receptor, our treatment is precisely aimed at the pathway disrupted by these mutations.”
Leading the way
Children’s National is the only site in the world doing this type of work, leading in pediatric endocrinology for novel clinical trials in growth disorders.
Funded by an investigator-initiated grant from BioMarin, this trial is the first-of-its-kind to treat children with genetic short stature who do not have achondroplasia. Other growth-related conditions included in this phase 2 trial were Noonan syndrome, NPR2 mutations and Aggrecan mutations.
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Andrew Dauber, MD, MMSc, chief of Endocrinology, and Denver Brown, MD, pediatric nephrologist, were featured as guests on The Kidney Chronicles: A Pediatric Nephrology Podcast. The episode focused on growth hormone in chronic kidney disease (CKD).
“As a pediatric nephrologist, it’s hard not to be interested in growth since growth failure and short stature is such a common finding in pediatric CKD, so I like to think all of us providers are interested in growth,” Dr. Brown said.
Both experts provided insight on common challenges of CKD patients, like when to start growth hormone treatment, how the condition can progress through metabolic acidosis and even the social impacts of common symptoms on everyday life. The episode gave a holistic view of how endocrinology and nephrology work together to support patients with CKD.
“While growth hormone is an important option, and I think an important treatment modality for kids who aren’t growing well with CKD, it’s just as important for families to think about other coping mechanisms for their kids,” Dr. Dauber said. “And that’s true for anyone with chronic disease.”
Listen to the full episode, “Growth hormone in CKD,” on PodBean or Spotify.
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The Children’s National Division of Endocrinology and Diabetes is excited to have 13 posters and presentations at the upcoming Pediatric Endocrine Society (PES) annual meeting in San Francisco, California. The range of topics being presented reflects the breadth of research happening within the division. Many of the posters and presentations continue diving deeper into research presented at conferences across the globe last year. (below)
Qualitative Interviews with Caregivers and Patients with Selected Genetic Causes of Short Stature, Raheem Seaforth, Niusha Shafaei, Kim Pitner, Roopa Shankar, Andrew Dauber
Clinical Spectrum of a New Overgrowth Syndrome Due To SPIN4 Dysfunction, Ruta Navardauskaite, Sujin Park … Youn Hee Jee
Vosoritide Treatment Improves Linear Growth and Absolute Bone Content and Density in Children with Hypochondroplasia: A 12-Month Prospective Study, … Ruta Navardauskaite, Anqing Zhang, Niusha Shafaei, Raheem Seaforth, Andrew Dauber
Vosoritide for Short Stature in Turner Syndrome: Interim results from a Prospective Single Center Trial, Roopa Shankar, Niusha Shafaei, Anqing Zhang, Niti Dham, Kim Pitner, Andrew Dauber
Lymphocytic Hypophysitis In An 8-Year-Old With De Novo Deletion at Chromosome 3Q13.31, Christina Lee, Chelsi Flippo
WNT/β-CATENIN Modulation by an Epigenetic Reader Gene, SPIN4, Sujin Park, Daniel Li, Yi-Wen Chen, Youn Hee Jee
Oral Abstract Sessions at PES 2026
Glycemic Variability and Arterial Stiffness in Youth-Onset Type 2 Diabetes: Interim Analysis of the Young at Heart Study, Nicole Rangos, Elizabeth Estrada, Stephanie Chung
A Phase 2 Basket Trial of Vosoritide in Children with Rasopathies, Acan and Npr2 Deficiency, Andrew Dauber, Anqing Zhang, Niusha Shafaei, Raheem Seaforth, Kim Pitner, Niti Dham, Roopa Shankar…
Navigating the Efficacy, Safety and Ethical implications of GLP-1 and SGLT-2 Therapies in Childhood Obesity and Type 2 Diabetes, Elizabeth Estrada
Other notable recent research from the division includes a new international guideline for genetic testing in children with short stature and how vosoritide affects growth signaling in patients with hypochondroplasia.
Below are the posters and presentations from members of our team at conferences last year. We’re excited for another year of research and collaboration with colleagues across the country and globe to improve the lives of children!
Severe Hypothyroidism due to Iodine Deficiency in an Infant with Short Bowel Syndrome Receiving Total Parenteral Nutrition, *Chaitanya Sambangi and Priya Vaidyanathan
Preliminary Assessment of Vosoritide for Short Stature in Turner syndrome: 6-month data from a Clinical Trial, Roopa Kanakatti Shankar, Niusha Shafaei, Anqing Zhang, Kim Boucher, Niti Dham, Andrew Dauber
European Society of Human Genetics
May 24-27, 2025, Milan, Italy
Invited Symposium Speaker
Novel Therapies for Growth Disorders: What Can We Expect from Personalised Medicine, Andrew Dauber
Variants Modulate Metformin-Liraglutide Pharmacokinetics and Glycemia in African American Youth Onset Type 2 Diabetes – The MIGHTY Study (link), … Stephanie Chung
Poster Presentations
Correlates of Device Use as Young Adults (YA) with Type 1 Diabetes leave Pediatric Care. (link), … Randi Streisand…
Short-term Reproducibility of I3C Risk Score in Predicting Cardiac Events in Young People, … Stephanie Chung
Incidence and Remission Rates of Youth-onset Type 2 Diabetes (Y-T2D) after Virtual Learning (VL) During the COVID-19 pandemic, Jody Grundman, Rachel Longendyke, Elizabeth Estrada, Stephanie Chung
Medical And Demographic Correlates of Session Attendance Among Historically Minoritized Youth with T1D in A CGM Behavioral Intervention (link), Amanda Perkins, Emma Straton, Christine Wang, John Barber, Letitia Gallant, Shideh Majidi, Randi Streisand
Social Determinants of Health Barriers in Newly Diagnosed Pediatric T1D and T2D (link), Jasmine Roberts, Sarah Holly, Roxanna Velasquez, Lauren Deanna, Diamond Patrick, Shideh Majidi
Social Determinants of Health Barriers—Differences between Public vs Private Insurance. (link), Jasmine Roberts, Sarah Holly, Roxanna Velasquez, Lauren Deanna, Diamond Patrick, Shideh Majidi
Implementation of Depression Screening in Newly Diagnosed Pediatric Type 1 Diabetes Patients (link), Jasmine Roberts, Sarah Holly, Lauren DeAnna, Diamond Patrick, Shideh Majidi
Latin American Society for Pediatric Endocrinology (SLEP) Annual Meeting
Clinical Predictors of Response to Growth Hormone in Idiopathic Short Stature, Andrew Dauber
A Pediatric Endocrinologist’s Guide to the Genetics of Growth, Andrew Dauber
Defects in PAPP-A2 Function: From Bedside to Bench and Back Again, Andrew Dauber
The 6th Pediatric Update Conference
September 25-26, 2025, Cairo, Egypt (delivered virtually)
Invited Symposium Speaker
Genetic Insights into Precocious Puberty, Andrew Dauber
Universidad San Francisco de Quito 4th biennial meeting
October 22-23, 2025, Quito, Ecuador
Invited Symposium Speaker
Treatment of Other Skeletal Dysplasias, Andrew Dauber
IPSAD: International Society for Pediatric and Adolescent Diabetes
November 5-8, 2025, Montreal, Canada
Posters without oral presentation
A New Model for Complex Type 1 Diabetes (T1D) Management: Feasibility of the Blossom Clinic Approach, Jody Grundman, Mai Tran, Lauren Clary, Lauren DeAnna, Roxanna Velasquez, Amanda Perkins
Assessing the Impact of Training and Mentorship in Liberia at Life for a Child (LFAC)-supported Clinics Transitioning Patients to Glargine Insulin, Amanda Perkins…
Social Determinants of Health (SDOH) and Demographic Disparities in Diabetic Ketoacidosis (DKA) Presentation at Type 1 Diabetes (T1D) Diagnosis, Sarah Holly, Jasmine Roberts, Jody Grundman, Rachel Longendyke, Shideh Majidi
A Medically Supportive Food Delivery Intervention for Children with Prediabetes or Type 2 Diabetes and Food Insecurity: Six Month Follow-up Results, Jasmine Roberts,… Shideh Majidi
Blossoming Outcomes: A Multidisciplinary Model for Youth with Type 1 Diabetes and Sub-optimal Glycemic Control, Jody Grundman, … Lauren Clary, Lauren Deanna, Amanda Perkins
Implementing an Early Screening Workflow for Stage 1 and Stage 2 Type 1 Diabetes in a Pediatric Endocrinology Clinic, Sarah Holly, Fran Cogen, Jody Grundman, Letitia Gallant, Shideh Majidi
Session Chair
Health Equity and Social Determinants of Diabetes, Shideh Majidi
Panel Speaker
Addressing Gaps in Diabetes Care Panel, Shideh Majidi
https://innovationdistrict.childrensnational.org/wp-content/uploads/2026/03/Endo-conferences-feature.jpg300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2025/09/InnovationDistrict_CN_WebHeader-1396px-1030x151.pngInnovation District2026-03-04 11:18:192026-08-05 12:00:38Children’s National at PES 2026 and a look back at conference research presentations in 2025
An international team of experts has published a new consensus guideline on genetic testing in children with short stature, providing an important resource for clinicians worldwide.
An international team of experts has published a new consensus guideline on genetic testing in children with short stature, providing an important resource for clinicians worldwide.
The guideline, published in the European Journal of Endocrinology, is the first of its kind and has received formal endorsement from several leading professional organizations, including the European Society of Pediatric Endocrinology (ESPE), Pediatric Endocrine Society (PES), Latin American Society of Pediatric Endocrinology (SLEP), Chinese Society of Pediatric Endocrinology and Metabolism (CSPEM), Japanese Society for Pediatric Endocrinology (JSPE), European Society of Human Genetics (ESHG), Japan Society of Human Genetics and Human Genetics Society of Australasia.
“Over the last decade, there have been tremendous advances in our understanding of the genetic factors that influence growth and stature,” said Andrew Dauber, MD, MMSc, chief of Endocrinology at Children’s National Hospital and lead author. “Many studies have shown that children with significant short stature frequently have undiagnosed genetic conditions.”
The big picture
The guideline was initiated and sponsored by the Clinical Practice Committee of the ESPE. A steering committee developed the guideline framework and invited experts to participate in the International Growth Genetics Guideline Consortium (IGGGC). The consortium includes pediatric endocrinologists, medical geneticists, clinical laboratory geneticists and a clinical epidemiologist/endocrinologist.
Two IGGGC subcommittees conducted a systematic review and literature search addressing two key clinical questions:
What is the diagnostic yield of genetic testing in children or adolescents with short stature in a non-selected population of short children or in populations selected for an additional clinical feature?
Will genetic testing in short stature improve the outcomes of individual patients?
Based on the available evidence, the authors developed consensus recommendations and diagnostic algorithms tailored to specific clinical presentation.
Children’s National leads the way
Dr. Dauber’s leadership has been central to this international effort. Researchers at Children’s National played a significant role in creating some of the evidence upon which this guideline was based. The Growth Disorders Program has been using this approach in clinical practice and is on the leading edge of delivering precision medicine for genetic growth disorders.
“We hope that genetic testing will become a more routine part of the evaluation of children with short stature,” said Dr. Dauber. “This will open up opportunities for precision medicine in the future.”
Additional authors from Children’s National include: Youn Hee Jee, MD.
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Children’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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https://innovationdistrict.childrensnational.org/wp-content/uploads/2026/01/NatRep-ID-Images_400-x-3003.jpg300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2025/09/InnovationDistrict_CN_WebHeader-1396px-1030x151.pngInnovation District2026-01-12 11:22:572026-08-05 11:59:34From research to reality: Transforming Turner Syndrome care at Children’s National
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.
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
https://innovationdistrict.childrensnational.org/wp-content/uploads/2025/12/2025-with-lightbulb-CNRI.jpg385685Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2025/09/InnovationDistrict_CN_WebHeader-1396px-1030x151.pngInnovation District2025-12-24 15:07:522026-07-08 10:45:30The best of 2025 from Innovation District
In 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.
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)
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)
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)
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)
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)
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)
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)
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)
Ashley Maxie-Moreman, PhD, clinical psychologist, spoke to ABC7 about what AI psychosis is and what parents need to know. (ABC7)
https://innovationdistrict.childrensnational.org/wp-content/uploads/2025/12/Logo-Collage-2025-feature.jpg300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2025/09/InnovationDistrict_CN_WebHeader-1396px-1030x151.pngInnovation District2025-12-23 13:53:592026-07-08 10:45:24Children’s National in the News: 2025
A 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.”
Other Children’s National authors include Andrew Dauber, MD, MMSc, Anqing Zhang, PhD, Niusha Shafaei, MSc, Kimberly Pitner, MSHS, BSN, RN, Raheem Seaforth.
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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.”
https://innovationdistrict.childrensnational.org/wp-content/uploads/2025/10/blood-glucose-monitoring-feature.jpg300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2025/09/InnovationDistrict_CN_WebHeader-1396px-1030x151.pngInnovation District2025-10-31 11:23:052025-10-31 11:24:47Improving research design in pediatric psychology
Children’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:
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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.”
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.
https://innovationdistrict.childrensnational.org/wp-content/uploads/2025/05/poster-presentation-at-PES-feature.jpg300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2025/09/InnovationDistrict_CN_WebHeader-1396px-1030x151.pngInnovation District2025-05-23 13:20:272026-01-09 16:01:51Encouraging early results for vosoritide in Turner syndrome
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.
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:
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”
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.
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.
Panelists discuss innovation during REI Week 2025.
Global Health Initiative community engagement event during REI Week 2025.
Chris Rees presents his REI Week 2025 lecture.
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.
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.
Ambroise Wonkman poses for a picture with Children’s National staff.
Tanzeem Choudhury presenting during REI Week 2025.
https://innovationdistrict.childrensnational.org/wp-content/uploads/2025/04/REI-Week-2025-Monday-Poster-Session-CNRI.jpg385685Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2025/09/InnovationDistrict_CN_WebHeader-1396px-1030x151.pngInnovation District2025-04-22 10:31:052026-09-21 15:10:01REI Week 2025 empowers the future in pediatric research and innovation
https://innovationdistrict.childrensnational.org/wp-content/uploads/2025/02/Article_Image_400x300_Dauber.jpg12501667Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2025/09/InnovationDistrict_CN_WebHeader-1396px-1030x151.pngInnovation District2025-02-19 13:05:102026-03-19 13:22:44Podcast: Growing hope: Advancing care for kids with growth disorders
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.
https://innovationdistrict.childrensnational.org/wp-content/uploads/2025/01/Dauber-at-computer-feature.jpg300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2025/09/InnovationDistrict_CN_WebHeader-1396px-1030x151.pngInnovation District2025-01-14 12:53:272025-01-14 12:54:58Breaking barriers in growth disorder treatment for families
Children’s National in Washington, D.C., was once again ranked as a top hospital in the nation by the U.S. News & World Report 2026-27 Best Children’s Hospitals 
The Children’s National Division of Endocrinology and Diabetes is excited to have 13 posters and presentations at the upcoming Pediatric Endocrine Society (PES) 
Children’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.
In 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.
A new 
Children’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.
