The decision came despite earlier concerns by FDA officials that the research data supporting the gene-therapy treatment for Duchenne was lacking.
or genetic conditions like Duchenne muscular dystrophy, there is little doctors can do to slow or treat the condition other than trying to manage symptoms, since only addressing the genetic changes responsible can help halt the disease.
Most people born with the inherited genetic disorder start to show symptoms of muscle weakness as early as age 2, and as the disease worsens, typically lose their ability to walk and eventually to breathe on their own. There is no cure, but doctors can prescribe medications to improve muscle strength, particularly for the heart and lungs, or recommend surgery to treat severe contractions that can affect posture to help patients live to their 20s or 30s.
In the first human studies of the gene therapy, in four boys ages 4 to 6 years old in 2018, the new dystrophin gene found its targets and the amount of healthier dystrophin they produced was “higher than we anticipated,” says Mendell. “We are at the five year mark for those kids and they haven’t declined; that’s very encouraging.”In the next, larger study of patients however, the results were less conclusive.
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