A 12-year-old boy with a severe form of muscular dystrophy has said it is "cruel" that a new drug is not being made available ...

The company raised $200 million after early findings hinted its therapy could be more potent than others like it, including ...

Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is ...

A Massachusetts man became the first person to try intranasal foralumab under an expanded access program for people with ...

Despite the obstacles, Riley Herrera had a life filled with family, sports, travel, faith and hope before his death on Nov.

While Illinois joins states challenging cuts to medical research funding, an Illinois State University lab supported by the ...

The Next Chapter is keeping the tears coming this season. Leah Messer is having a tough time getting through to daughter Ali ...

CEO Dr James Garner says at the time of the December 18 trial bombshell that knocked 85% off the value of Percheron shares, ...

Five oculopharyngeal muscular dystrophy (OPMD) patients have now been treated with the gene therapy candidate BB-301 in a ...

A seven-year-old boy from Dublin is on his way to California to undergo testing after being diagnosed with a rare condition last year.

Some of the market’s biggest winners start off as the smallest players. While trillion-dollar tech giants like the ...