President Trump says his administration pledges “to protect the American heart and to keep the American people safe, strong, healthy, and free.” Official Message from President Donald J.…
Read MoreGenetic Condition
FDA Approves Gene Therapy for Treating Spinal Muscular Atrophy
Patients of all ages now have access to care for the rare genetic disease. The Food and Drug Administration (FDA) has announced approval for Itvisma (onasemnogene abeparvovec-brve), which…
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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…
Read MoreFDA 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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