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 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 ...

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 ...

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 ...

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 ...

Myofibrillar myopathy type 6 (MFM6) is a rare genetic muscle disorder that leads to severe muscle weakness and a drastically ...

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