Genetic Diseases, Inborn

Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping

A Long-term Follow-up Study in Participants Who Received CTX001

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

Health Economic Evaluation of Non-Invasive Prenatal Exclusion Diagnosis

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

Prenatal Transplantation for Fetuses With Fanconi Anemia

A Study to Learn About the Safety and Efficacy of the Drug Oleogel-S10 in Japanese Patients With Epidermolysis Bullosa

A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia

A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients

Effect of Benralizumab in Atopic Dermatitis

Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia

A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

Effect of Large Neutral Amino Acids in Adults With Classical Phenylketonuria

Evaluating Prenatal Exome Sequencing Study

Pilot Study of Cardiac MR in Patients With Muscular Dystrophy

Guanfacine Extended Release for the Reduction of Aggression and Self-injurious Behavior Associated With Prader-Willi Syndrome

Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors

NYCKidSeq: Incorporating Genomics Into Clinical Care of Diverse NYC Children