Sarepta has stopped developing SRP-5051, its exon 51-skipping therapy for Duchenne MD, due to long-term safety concerns and ...

The abstract will be made available on the publications section of the Capricor website.

EBR Systems is among ASX healthcare stocks with upcoming catalysts, expecting feedback on a FDA substantive review expected ...

Pratteln, Switzerland, November 12, 2024 – Santhera Pharmaceuticals (SIX: SANN) announces the signing of an exclusive supply agreement with Ali Al Suwaidi Trading Est. (ASTE) for the treatment of ...

In particular, the company's main value drivers address myotonic dystrophy type 1 [DM1], Duchenne muscular dystrophy [DMD], ...

Gain insight on Rigel Pharmaceuticals, AbbVie and more in the latest Market Talks covering the Health Care sector.

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 ...