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Making progress with a gene therapy for muscular dystrophy – Advanced Science News

Wednesday, 25 January, 2023

Making progress with a gene therapy for muscular dystrophy – Advanced Science News

Duchenne muscular dystrophy (DMD) is a fatal condition caused by a single gene mutation on the X-chromosome — being X-linked means only males suffer from the disease. Roughly 1 in 5000 males are born with this condition and there is currently no cure, with the median age of survival 23 years.

Gene therapies are a promising treatment option, and a recent study published in Science Translational Medicine describes success with one such therapy using an animal model. These results have paved the way for ongoing human trials, which have shown a promising ability of this therapy to slow the progression of the disease. Pharmaceutical companies see the value too, with one company, Sarepta, expecting approval of a Duchenne muscular dystrophy gene therapy as early as June of this year.

Read more at: https://www.advancedsciencenews.com/making-progress-with-a-gene-therapy-for-muscular-dystrophy/

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