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Genetic Disorders

Anemia Familial Insomnia Recessive Dystrophic Epidermolysis Bullosa (RDEB) Severe Combined Immunodeficiency (SCID) Spinal Muscular Atrophy Niemann-Pick Disease SMA Type 1 Methylmalonic Acidemia Hereditary Angioedema Muscle and Bone Development Disorders Barth Syndrome ADA-SCID Mitochondrial Diseases Russell-Silver Syndrome Heart Defects

NEJM Study Finds 95% Long-Term Success for ADA-SCID Gene Therapy

New long-term results validate a path to broader access through cryopreserved manufacture.

FDA Grants Accelerated Approval to Forzinity, First Therapy for Barth Syndrome

FDA Approves Ionis’ Dawnzera to Prevent Hereditary Angioedema Attacks