Sarepta Therapeutics (Nasdaq: SRPT) has announced it will cease development of SRP-5051, an experimental drug for Duchenne ...

Filippis is currently the chief operating officer of Percheron Therapeutics (ASX:PER), which has an ongoing Phase IIb ...

including myotonic dystrophy type 1 (DM1), facioscapulohumeral muscular dystrophy (FSHD), and Duchenne muscular dystrophy (DMD). In DM1, Avidity Biosciences continues to progress with their Phase 3 ...

Analyst Joseph Schwartz from Leerink Partners maintained a Buy rating on Solid Biosciences (SLDB – Research Report) and keeping the price ...

WHAT IT'S ABOUT The documentary "The Remarkable Life of Ibelin" tells the story of Mats Steen, a Norwegian man with Duchenne ...

Sarepta Therapeutics is advancing SRP-9003 gene therapy for LGMD, with phase 3 study results expected in early 2025. Learn ...

For the first time this year, the students of McKinley Elementary aided the Muscatine Fire Department in raising money for its Muscular Dystrophy Association Boot Drive. As of Tuesday, they have ...

An FDA expansion of Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) Elevidys has sparked a new surge in sales for the ...

SRP-5051 is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations amenable to exon 51 skipping. With this ...

Sarepta will discontinue its next generation of exon-skipping drugs for Duchenne muscular dystrophy over safety concerns, in ...

Elevidys sales have increased since the FDA made a controversial choice to expand the therapy's use. Meanwhile, Sarepta is ...