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 ...

Medera Inc. ("Medera"), a clinical-stage biopharmaceutical company developing next-generation cardiovascular therapeutics, ...

Roche is launching a new global phase 3 trial for the controversial Duchenne muscular dystrophy gene therapy Elevidys in ...

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 ...

Andelyn Biosciences, a leading and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced today a partnership with Evolyra Therapeutics to ...

Sarepta Therapeutics (SRPT) stock and Roche (RHHBY) stock are in focus as Duchenne therapy Elevidys faces a new late-stage ...

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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 ...

Emma Ciafaloni, MD, FAAN, dives into the latest developments in gene therapy for muscular dystrophies, focusing on Duchenne muscular dystrophy, and discusses challenges, genetic causes, and the ...

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 ...

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 ...