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Rare Diseases

Neurological Disorders Customized Treatments Biliary Malformations Diagnosis of Rare Conditions Barth Syndrome Treatment Innovative Treatments Immune System Disorders Innovative Therapies Infection Vulnerability Metabolic 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.

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