Crispr has already secured a historic first approval for a CRISPR/Cas9 drug and has several more potential blockbuster ...

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 ...

The company has shown promising results in its preclinical and early clinical trials, particularly with its lead program ...

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 ...

A Pennsylvania woman, 38, tells PEOPLE that she credits Dateability, a dating app focused on the disabled and chronically ill ...

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 ...

Zolgensma gene therapy is the most effective in infants with SMA who are presymptomatic and 6 weeks old or younger, according ...

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 ...