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News at a glance: Muscular dystrophy therapy, lab-grown chicken, and humans ’ toll on wildlife
BIOMEDICINE Muscular dystrophy therapy approved The U.S. Food and Drug Administration has approved the first gene therapy for Duchenne muscular dystrophy (DMD), a genetic disease that cripples boys and usually results in death by age 30. The treatment from Sarepta Therapeutics introduces a short version of the gene for dystrophin, a crucial muscle protein, which is mutated in patients with DMD. A one-time intravenous infusion of a virus delivers the functioning “microdystrophin” gene into patients’ muscle cells. The 22 June approval is only for boys 4 to 5 years old, a group that appeared likely to ben...
Source: Science of Aging Knowledge Environment - June 29, 2023 Category: Geriatrics Source Type: research