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Rare Diseases

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