Annual event brings together 1,200 plus families, physicians, researchers, caregivers, industry partners, and those living with Duchenne and Becke ...

In this week’s edition of InnovationRx, we look at the dangers of RFK Jr.’s vaccine advisory committee, how Trump’s visa ban ...

In another tragic incident, Sarepta Therapeutics (NASDAQ: SRPT) saw a second death in connection with its Duchenne muscular ...

I am upgrading Dyne Therapeutics stock to a 'Buy' rating due to promising late-stage DM1 and DMD drug candidates and ...

Many parents said they were afraid of noninvasive ventilation, with some citing inaccurate concerns about ventilation devices.

Another patient has died from acute liver failure after receiving Sarepta’s gene therapy for DMD ; After a quiet start to the ...

"There were tears everywhere," Hana Herrick, school services coordinator at Lurie Children’s Hospital, tells PEOPLE ...

The boys live in Reed City with their mother Tabatha; father Michael, who is the Osceola County Road Commission manager and ...

Capricor Therapeutics (NASDAQ:CAPR) received the Orphan Drug designation from the U.S. FDA for its lead cell therapy candidate, Deramiocel, to treat becker muscular dystrophy. With its orphan drug ...

Capricor Therapeutics has received an orphan drug designation for its drug aimed at potentially treating Becker Muscular Dystrophy. The biotechnology company said Tuesday that the Food and Drug ...

Ticker: Second patient death reported with gene therapy for muscular dystrophy; Salmonella poisoning linked to pistachio ...

How the U.S. FDA might respond became a serious question for Wall Street as Sarepta Therapeutics Inc. made known a second death due to acute liver failure with gene therapy Elevidys (delandistrogene ...