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/
