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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 ...

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 ...

Sarepta Faces Investor Skepticism As Duchenne Data Falls Short Of Statistical Significance

Sarepta's Duchenne trial missed its main goal but showed trends favoring treatment; Q3 earnings topped estimates amid ...
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 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 ...

Sarepta Therapeutics shares plunge after disappointing trial data

Sarepta Therapeutics shares plunged 37% after two Duchenne muscular dystrophy drugs failed a key trial, raising regulatory ...

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 ...
PharmaTimes

Santhera reports promising long-term data for AGAMREE in Duchenne muscular dystrophy

Santhera Pharmaceuticals has announced positive topline results from a long-term analysis of AGAMREE (vamorolone) in patients ...

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 ...

Charlie Plummer, Stephen Graham, Toni Collette, Isabela Merced, Maisy Stella & Anthony Hopkins Set For ‘Ibelin’ From Vendôme Pictures & Pathé – AFM

L.A. and Paris-based Vendôme Pictures, producer of Oscar Best Picture winner Coda, has unveiled the cast for previously ...

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 Stock Dumps: Pharma Bro Martin Shkreli Still Likes It

Sarepta Therapeutics, Inc. (NASDAQ: SRPT) stock fell sharply Tuesday following disappointing results from a key clinical trial and ongoing concerns about its drug pipeline. However, "Pharma Bro" ...