The U.S. Food and Drug Administration has approved the first gene therapy for the treatment of ambulatory pediatric patients with Duchenne muscular dystrophy (DMD).
The recombinant gene therapy Elevidys is applicable to chldren in the age group of 4 to 5 having this disease with a confirmed mutation in the DMD gene who do not have a pre-existing medical reason preventing treatment with this therapy.
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by Joji Xavier
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