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Rare Diseases

Childhood Autoimmune Disorders Leber Congenital Amaurosis Hunter Syndrome Immune Disorders Treatment Options Spinal Muscular Atrophy SCN2A Gene Disorders

FDA Grants Accelerated Approval to Kresladi, First Gene Therapy for Severe Pediatric LAD-1

The one-time autologous treatment won conditional clearance on biomarker gains, requiring long‑term confirmation of benefit.

Lilly Licenses MeiraGTx Eye Gene Therapy in Deal Worth Up to $475 Million