Nationwide Children’s Research Benefits Kids Everywhere
Nationwide Children’s Research Benefits Kids Everywhere https://pediatricsnationwide.org/wp-content/uploads/2024/04/020524BT517_FeatureHR-crop-1024x619.jpg 1024 619 Nationwide Children's https://secure.gravatar.com/avatar/8087ae3f73e51e24df5819a3363b55e0?s=96&d=mm&r=g
Pediatric research benefits children everywhere. Through the stories below, discover more about the breadth and depth of research at Nationwide Children’s that is improving child health.
Optimizing Prenatal and Neonatal Care for Infants With Treatable Rare Diseases
Not long ago, a diagnosis of molybdenum cofactor deficiency (MoCD) type A meant death before kindergarten.
Since the FDA approval of NULIBRY® (fosdenopterin) in 2021, however, children diagnosed with MoCD type A — an inherited metabolic disorder that results in severe developmental delay and neurological damage due to the accumulation of sulfites and S-sulfocysteine (SSC) in the brain — have hope for a significantly longer life.
If this intravenous (IV) drug is administered prior to serious brain injury, they may avoid significant intellectual and physical disability.
The trick? Getting the diagnosis and beginning treatment before significant damage has been done.
Meet Ghaith: Making History for MoCD
Baby Ghaith was born via an uncomplicated C-section on March 29, 2023. He received his first IV dose of fosdenopterin before he was 10 minutes old. This administration within minutes of delivery is the earliest treatment of MoCD known to the Nationwide Children’s team.
A New Future for Boys with Duchenne Muscular Dystrophy (DMD) Thanks to Gene Therapy
Connor's and Easton's Stories
Connor and Easton both participated in an early clinical trial for gene therapy for DMD. Find out how they are doing 5 years after receiving the gene therapy.
Gideon's Story
Meet Gideon, the first patient to receive gene therapy for DMD outside of a clinical trial at Nationwide Children’s.
Learn More About Research at Nationwide Children’s and the Families That Benefit
Ohio Cancer Research Recognizes Nationwide Children's Hospital
Learn more about how inspirational leadership advances research.
Genome Sequencing Provides Answers
Meet Summer. Her illness started shortly after birth, but it took nearly her whole childhood to find answers. In 2020, the genomics team finally gave her family a name for her disorder.
Advancing Regenerative Medicine
Meet the physician-scientist at the forefront of tissue engineering.
Making Progress in Cardiovascular Research
Meet the scientists advancing research for children with heart disease.
Integrating Research and Clinical Care for Children With Debilitating Pancreatitis
Meet the pioneering researcher behind islet cell transplantation.
Complex Feeding Disorders: Integrating Research and Clinical Care
Meet Hayes, who went from a complex eating disorder to eating like a champ with the help of the Infant Feeding Disorders Program.
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