Sarepta Duchenne drugs come up short in confirmatory test

Still, CEO Doug Ingram claimed removing the two drugs, Vyondys 53 and Amondys 45, from market would make “little sense” and ...
Fierce Pharma

Sarepta pushes for full approval of Duchenne drugs despite missed confirmatory trial goal

A long-running confirmatory trial of two of Sarepta Therapeutics’ Duchenne muscular dystrophy drugs has missed its main goal.

Sarepta's shares plunge after trial for muscle-wasting disease drugs miss key goal

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

Sarepta shares slide as DMD treatment woes pile up

Shares in Sarepta Therapeutics lost nearly 40% of their value after the company said a trial designed to confirm the efficacy ...

Sarepta's Duchenne gene therapy misses main goal in late-stage study; shares fall

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

Sarepta’s DMD Exon-Skippers Fail Confirmatory Study, Stock Craters

Sarepta nevertheless plans to push for full FDA approval of Vyondys 53 and Amondys 45 based on what it said are “encouraging ...

Sarepta slumps as gene therapy setback adds to drug pipeline woes

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

Sarepta’s stock plummets as disappointing trial data raise questions on DMD drug’s success

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

Parent's plea for muscular dystrophy drug for son

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

Sarepta slumps as trial failure deepens woes for drugmaker

Sarepta Therapeutics' shares plunged nearly 40% in premarket trading on Tuesday as failure of two of its approved drugs for a ...
MedPage Today on MSN

Long-Term Givinostat Data Reinforce Safety Profile in Duchenne Muscular Dystrophy

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

Two of Sarepta’s DMD drugs fail to meet primary endpoint in confirmatory trial

Two of Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) candidates have failed to reach their primary endpoint in a Phase III confirmatory trial. In the ESSENCE study, AMONDYS 45 (casimersen) ...