Reproductive Capacity and Iron Burden in Thalassemia

Pediatric Precision Laboratory Advanced Neuroblastoma Therapy

Farber Disease Natural History Study

Study to Assess the Effect of Long-term Treatment With GBT440 in Participants Who Have Completed Treatment in Study GBT440-031

A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia

Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias

Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)

Personalized Research on Diet in Ulcerative Colitis and Crohn's Disease

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II

Improving Preterm Infant Outcomes With Family Integrated Care and Mobile Technology

Pediatric Obesity Weight Evaluation Registry (POWER) Study

Safety Of Rivipansel (GMI-1070) In The Treatment Of One or More Vaso-occlusive Crises In Hospitalized Subjects With Sickle Cell Disease

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I

PAZIT Study for Children and Young Adults With Relapsed or Refractory Sarcoma

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Subjects With Transfusion-Dependent β-Thalassemia, Who Have a β0/β0 Genotype

Clinical Utility of Pediatric Whole Exome Sequencing

Open‐Label Study of Long‐Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann‐Pick Disease Type C

Efficacy and Safety of Rivipansel (GMI-1070) in the Treatment of Vaso-Occlusive Crisis in Hospitalized Subjects With Sickle Cell Disease

Phase II Study of Binimetinib in Children and Adults With NF1 Plexiform Neurofibromas

A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

Neuroblastoma Maintenance Therapy Trial

Natural History of Rett Syndrome & Related Disorders

Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMT CTN 1507)

A Study of IMR-687 in Adult Patients With Sickle Cell Anaemia (Homozygous HbSS or Sickle-β0 Thalassemia)

A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)

Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007