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 ...

Achieved the primary objective with favorable safety and tolerability, no discontinuations and no serious adverse events -- - ...

Capricor Therapeutics has accused Nippon Shinyaku and its U.S. subsidiary of botching the rollout of a Duchenne muscular ...

Eight therapies are approved for Duchenne muscular dystrophy (DMD) in the U.S., and all have been approved since 2016. "It took 30 years from the discovery of the gene associated with Duchenne ...

In an eagerly anticipated decision, the Food and Drug Administration Thursday approved the first gene therapy for muscular dystrophy. "Today's approval addresses an urgent unmet medical need and is an ...

Experts discuss the landscape of treatment options for Duchenne muscular dystrophy, examine the benefits and risks of gene therapy and conventional steroid treatments, and emphasize the need for early ...

Givinostat will be made available to eligible patients with Duchenne muscular dystrophy after its manufacturer struck a ...

Capricor Therapeutics (CAPR) is down ~11% in Friday trading after announcing following Thursday's market close it is suing ...

BELIEVE WHAT HAPPENED NEXT. FOUR YEAR OLD LUCAS LOVES A GOOD PLAYDATE AT THE PARK. HI, LUCAS. HI. IT’S SANDBOX TRUCKS TODAY, BUT HIS FAVORITE PLAYING SUPERHEROES. THIS IS HIS FAVORITE RIGHT NOW. PJ ...

In human biology, the protein dystrophin is a shining example of Joni Mitchell’s classic line, “you don’t know what you’ve got ’til it’s gone.” Dystrophin stabilizes muscle cells. In its absence, the ...

Dyne Therapeutics’ experimental treatment for Duchenne muscular dystrophy (DMD) showed promise in new data from a small Phase 1/2 trial dubbed DELIVER, the pharma announced this week. The drug, ...

Today’s final draft guidance says givinostat (also called Duvyzat and made by ITF Pharma) can be used to treat Duchenne muscular dystrophy (DMD) in people aged 6 and over who are able to walk or stand ...