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Genetic Disorders

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

Sanofi’s Efdoralprin Alfa Beats Standard Therapy in Phase 2 AATD Trial

The recombinant design signals a possible plasma-free option that could ease weekly treatment burdens.

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

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

FDA Approves Ionis’ Dawnzera to Prevent Hereditary Angioedema Attacks