() -Shares of Sarepta sank 36% before the bell on Tuesday after a trial for two gene-targeted therapies for a muscle-wasting ...

Sarepta stock crashed late Monday after the biotech company said two of its muscular dystrophy drugs failed their ...

Sarepta said on Monday that its late-stage study testing two gene-targeted therapies for Duchenne muscular dystrophy did not ...

Shares of Sarepta Therapeutics Inc. plunged in extended trading Monday after the biotechnology company disclosed ...

Givinostat is an oral histone deacetylase (HDAC) inhibitor approved by the FDA in 2024 to treat Duchenne patients ages 6 ...

His parents Stephen and Jenny have asked Sheffield Children's NHS Foundation Trust to approve the use of drug givinostat, ...

The Swiss pharma is paying $12 billion for Avidity Biosciences and its three late-stage antibody oligonucleotide conjugates.

Sarepta Therapeutics ( SRPT) shares fell after its late-stage trial for two gene therapies, AMONDYS 45 (casimersen) and VYONDYS 5 (golodirsen), aimed at treating Duchenne muscular dystrophy, did not ...

Data from the Phase Ia/b study demonstrated that SAT-3247 was well-tolerated and safe with a desirable PK profile.

While it’s established that Duchenne muscular dystrophy (DMD) causes a loss of muscle mass and strength, few studies have examined the gastrointestinal issues tied to DMD. Initial fractures are the ...

Find out if it’s time to ask about your child’s symptoms — and if they could be a sign of Duchenne. Duchenne muscular dystrophy — sometimes referred to simply as Duchenne — is a genetic disorder that ...