Shares of Sarepta Therapeutics SRPT plunged nearly 24% last week after the company reported the death of a patient following ...

can now be used to treat both ambulatory and non-ambulatory boys aged four and above with DMD, a major expansion on its earlier accelerated approval with a very narrow label covering ambulatory ...

Satellos Bioscience Inc. (TSX: MSCL, OTCQB: MSCLF) ("Satellos" or the "Company"), a clinical-stage biotechnology company developing life-improving medicines to treat degenerative muscle diseases, ...

The patient, whom Sarepta described as a young man and later told news outlets was 16 years old, suffered acute liver failure ...

Study results support the role of functional dystrophin and suggest that delandistrogene moxeparvovec stabilizes or slows ...

One patient with Duchenne muscular dystrophy (DMD) has passed away following treatment with Sarepta Therapeutics’ gene therapy Elevidys, the biotech ...

About the CONNECT Clinical Program CONNECT1-EDO51 is an open-label, multiple ascending dose ... two cohorts of boys and young men living with DMD amenable to exon 51 skipping and its endpoints ...

After some earlier data sparked questions from analysts, Avidity Biosciences has released more data for its RNA-based ...

The FDA has granted Priority Review to the BLA for deramiocel (CAP-1002) for the treatment of Duchenne muscular dystrophy cardiomyopathy.