The first gene therapy for children with Duchenne muscular dystrophy has been approved by the U.S. Food and Drug Administration. The therapy can be used in 4- and 5-year-olds with the degenerative ...

Astellas Pharma has done some spring cleaning of its pipeline, dropping two phase 1 candidates and slamming the brakes on a ...

Investigational deramiocel is back under FDA review again ...

The first gene therapy for a deadly form of muscular dystrophy received preliminary U.S. approval on Thursday despite concerns from some government scientists about the treatment's ability to help ...

Without finishing its Phase 3 clinical trial (usually, there are three phases), Sarepta sought a “fast-track” via conditional approval for a limited population and ultimately hoped to be able to treat ...

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The FDA's recent expanded approval of delandistrogene moxeparvovec (Elevidys) widened access to the gene therapy to include ambulatory and non-ambulatory Duchenne muscular dystrophy patients ages 4 ...

For 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 ...