JPMorgan sees a long-term buying opportunity after Sarepta (SRPT) shares were under pressure last week post earnings. Near-term, if one ...

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

Neurotech says Dr Anthony Filippis will leverage partnering success and capital markets expertise to progress its NTI164 ...

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 ...

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 ...

Belief Biomed Inc.’s gene therapy drug BBM-D101 has been awarded U.S. orphan drug and rare pediatric disease designations by the FDA for the treatment of Duchenne muscular dystrophy (DMD).

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 Therapeutics has ended development of a late-phase Duchenne muscular dystrophy (DMD) drug candidate over a safety problem. The safety signal deprived Sarepta of a near-term shot at accelerated ...