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…
Read MoreMuscular Dystrophy
FDA Adds Warning and Restrictions to Duchenne Gene Therapy
Updated labeling limits treatment to ambulatory children after reports of fatal liver failure in non-ambulatory patients. The U.S. Food and Drug Administration (FDA) has approved significant labeling changes…
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FDA Approves Duchenne Muscular Dystrophy Treatment for Children
Duvyzat receives approval under fast-track designation. The U.S. Food and Drug Administration (FDA) has approved Duvyzat (givinostat) as an oral medication for the treatment of Duchenne Muscular Dystrophy…
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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