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 ...

Sarepta Therapeutics ' gene therapy to treat Duchenne muscular dystrophy (DMD), a progressive muscle-wasting disorder, failed to meet the main goal of a late-stage trial when tested in patients ...

EVRY, France--(BUSINESS WIRE)--Atamyo Therapeutics, a biotechnology company specializing in the development of next-generation gene therapies for limb-girdle muscular dystrophy (LGMD), announced at ...

When I was diagnosed with Duchenne muscular dystrophy 20 years ago, there was no hope. The guidance the diagnosing doctor gave my parents was simple: Love your child as much as you can now because he ...

Roche Holding said it would start a late-stage study for a gene therapy for Duchenne muscular dystrophy to seek approval in the European Union after drug regulators in the bloc turned it down last ...

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 ...

Investigational deramiocel is back under FDA review again ...

Roughly once a year in Switzerland, a family receives the devastating diagnosis that their child is affected by a severe form of muscle weakness. This hereditary disease, known as LAMA2-related ...

Left: Muscle fibers expressing dysferlin (purple) made from gene-edited muscle stem cells transplanted into a mouse that lacks dysferlin. Right: Muscle fibers from the recipient mouse that are ...