FDA Approves First Treatment for Rare Disease

Ctexli offers hope for patients with a previously untreated metabolic disorder, Cerebrotendinous Xanthomatosis. The U.S. Food and Drug Administration (FDA) has approved Ctexli (chenodiol) as the first treatment…

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FDA Approves Gene Therapy for Muscular Dystrophy in Children

Treatment is intended for kids with a confirmed mutation of the DMD gene. The U.S. Food and Drug Administration (FDA) recently approved Elevidys, the first gene therapy for…

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