Patients Treated for SCID (1968-Present)

Natural History Study of SCID Disorders

Regional Distribution Differences Between Neurally Adjusted Ventilatory Assist and Pressure Support Ventilation

Adolescent Master Protocol for Participants 18 Years of Age and Older (AMP Up)

Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria

Cerebral Palsy Hip Outcomes Project - International Multi-centre Study

A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids

Biobanking of Rett Syndrome and Related Disorders

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

Elbasvir (EBR)/Grazoprevir (GZR) in Pediatric Participants With Chronic Hepatitis C Infection (MK-5172-079)

Study of Two Surgical Drilling Techniques to Treat Juvenile Osteochondritis Dissecans of the Knee

Patients Treated for Chronic Granulomatous Disease (CGD) Since 1995

The Treatment of Progressive Early Onset Spinal Deformities: A Multi-Center Outcome Study

A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)

FLT-PET Imaging of Brain Tumors in Children

Safety and Efficacy Study of Transcatheter Closure of Ostium Secundum ASDs

MIBG With Dinutuximab

Laboratory-Treated Peripheral Blood Cell Infusion After Donor Stem Cell Transplant in Treating Patients With Hematologic Cancers or Other Diseases

Genetic Study of Chronic Prostatitis/Chronic Pelvic Pain Syndrome (CP/CPPS)

Different Therapies in Treating Infants With Newly Diagnosed Acute Leukemia

Gene Mutations and Rescue in Human Congenital Diaphragmatic Hernia

Placebo Controlled Study of Baclofen for GERD in Children With Cerebral Palsy

Congenital Heart Disease GEnetic NEtwork Study (CHD GENES)

Neuroblastoma Biology Study

131I-MIBG Alone VS. 131I-MIBG With Vincristine and Irinotecan VS131I-MIBG With Vorinistat

A Research Study to Bank Samples for Future Evaluation to Identify Biomarkers That Predispose Crohn's Disease and Ulcerative Colitis Patients to Develop Hepatosplenic T-Cell Lymphoma (HSTCL)

Natural History of Rett Syndrome & Related Disorders

Genomic Basis of Neurodevelopmental and Brain Outcomes in Congenital Heart Disease (CHD Brain and Genes)

The Pediatric Anesthesia Quality Improvement Project

Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia

A Study to Test the Safety and Efficacy of the Drug Larotrectinib for the Treatment of Tumors With NTRK-fusion in Children

Use of the Ask Suicide-Screening Questions (ASQ) in Pediatric Outpatient Settings

Riociguat in Children With Pulmonary Arterial Hypertension (PAH)

Transfer Impedance in Cystic Fibrosis

Cholestasis Reversal: Efficacy of IV Fish Oil

Multicenter Prospective Cohort Study on Current Treatments of Legg-Calvé-Perthes Disease

Coagulation and Fibrinolysis in Pediatric Insulin Titration Trial (CAF-PINT)

Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease

Phase 1 Pediatric Pharmacokinetics/Pharmacodynamics (PK/PD) Study