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

Abdel Hamid Dbeibah, has agreed to prepare a national treatment program for muscular dystrophy. According to Dbeibah’s decision, which was published by the Ministry of Health on Sunday, the program is ...

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

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

Brian Skorney, an analyst from Robert W. Baird, maintained the Buy rating on RegenXBio (RGNX – Research Report). The associated price ...

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).

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 ...

The investigational therapy, vesleteplirsen, had been positioned as an updated version of Sarepta’s original exon 51-skipping ...

Duchenne Muscular Dystrophy (Duchenne) LYNX Phase 2 trial in boys with Duchenne ... along with data from the FOX trial of Duchenne children previously treated with gene therapy, to guide the design ...

Despite diagnostic and therapeutic advances, mortality and the multi-systemic health impact of SMA continue to be experienced ...