A Trial to Evaluate the Safety and Efficacy of Elamipretide in Subjects With Primary Mitochondrial Myopathy Followed by an Open-Label Extension

Pediatric Gastroparesis Registry

Innovative Approaches to Gauge Progression of Sturge-Weber Syndrome

Timing of Inguinal Hernia Repair in Premature Infants

Safety Study of Gene Modified Donor T-cells Following TCR Alpha Beta Depleted Stem Cell Transplant

Randomized Controlled Trial of Fetoscopic Endoluminal Tracheal Occlusion With a Balloon Versus Expectant Management During Pregnancy in Fetuses With Left Sided Congenital Diaphragmatic Hernia and Moderate Pulmonary Hypoplasia.

'TOTAL' (Tracheal Occlusion To Accelerate Lung Growth) Trial

OMEGA: Outcome Measures in Eosinophilic Gastrointestinal Disorders Across the Ages

SMOF Lipid for Children With Parenteral Nutrition Induced Liver Injury

Safety and Efficacy Study of Viaskin Peanut in Peanut-allergic Young Children 1-3 Years of Age

Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant

Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)

Quality Improvement Project - Evaluation of Current Standard of Care for Feeding Practices in the NICU

A 5-year, Prospective, Non-interventional, Multicenter Registry in Sickle Cell Disease (SCD) Patients

Targeted Protein Fortification in Extremely Low Birth Weight Preterm Infants

A Long-term Follow-up Study to Evaluate the Impact of Lumicitabine on the Incidence of Asthma and/or Wheezing in Infants and Children With a History of Respiratory Syncytial Virus Infection

Safety and Pharmacokinetic Study of Intravenous Ibuprofen in Pediatric Patients

A Study of Safety and Efficacy of MK-1986 (Tedizolid Phosphate) and Comparator in Participants From Birth to

A 5-year Longitudinal Observational Study of Patients With Nonalcoholic Fatty Liver (NAFL) or Nonalcoholic Steatohepatitis (NASH)

Acetaminophen Biomarkers