Pediatric Precision Laboratory Advanced Neuroblastoma Therapy

Evaluation of the Duration of Therapy for Thrombosis in Children

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

Study of DFMO in Combination With Bortezomib for Relapsed or Refractory Neuroblastoma

HeadStart4: Newly Diagnosed Children (

Standardized Protocol of Surgery and Radiation for Patients With Brain Metastases in Relapsed Neuroblastoma

Pre-Symptomatic Study of Intravenous AVXS-101 in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2

Registry Evaluating Premature and Term-Near-Term Neonates With Pulmonary Hypertension Receiving Inhaled Nitric Oxide

An Intermediate Expanded Use Trial of DFMO

Study of Nifurtimox to Treat Refractory or Relapsed Neuroblastoma or Medulloblastoma

Safety Study of Unlicensed, Investigational Cord Blood Units Manufactured by the NCBP for Unrelated Transplantation

Pediatric Obesity Weight Evaluation Registry (POWER) Study

Testing the Effect of Adding Chronic Oral Azithromycin to Inhaled Tobramycin in People With CF

Neuroblastoma Maintenance Therapy Trial

A Phase 2 Study of Ruxolitinib With Chemotherapy in Children With Acute Lymphoblastic Leukemia

CHaractErizing CFTR Modulated Changes in Sweat Chloride and Their Association With Clinical Outcomes

Post Approval Study of Liposorber LA-15 System for the Treatment of Focal Segmental Glomerulosclerosis in Children

Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy

Trial of Tolcapone With Oxaliplatin for Neuroblastoma

Multicenter Study of Patient-reported Gastrointestinal Symptoms in People With Cystic Fibrosis