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Genetic Disorders

Spinal Muscular Atrophy Sickle Cell Disease Gene Editing Cystic Fibrosis Leber Hereditary Optic Neuropathy Leber's Hereditary Optic Neuropathy Fibrodysplasia Ossificans Progressiva Hemophilia Hunter Syndrome

FDA Approves Isembyld, First Muscle‑Directed Therapy for Spinal Muscular Atrophy

Isembyld is an IV add‑on to existing SMN2 treatments that showed motor improvement in children and will begin U.S. shipments.

FDA Approves Regeneron’s Pasatru for Adults With FOP