Biotech reported positive interim data from a mid-stage trial of a treatment for the rare muscle disorder Duchenne muscular ...

A possible gene therapy approval and a series of major trials readouts set the stage for a busy year of drug development in ...

Duchenne muscular dystrophy, or DMD, is a severe genetic disorder, typically affecting males, and is characterized by progressive ...

Capricor announced it intends to file a BLA for deramiocel for treatment of patients with DMD cardiomyopathy. Click here to ...

Wave Life Sciences (WVE) stock rises on positive interim data from a Phase 2 trial for for WVE-N531, a therapy for Duchenne ...

Pratteln, Switzerland, September 24, 2024 – Santhera Pharmaceuticals (SIX: SANN) announces that Swissmedic, the Swiss Agency for Therapeutic Products, has accepted for review the marketing ...

Wave’s result is in line with the data that supported accelerated approval of NS Pharma’s exon 53 DMD drug Viltepso. NS ...

Wave Life Sciences Ltd. (NASDAQ:WVE) revealed interim data from the ongoing Phase 2 FORWARD-53 study of WVE-N531 for Duchenne ...

The embryos were female, and Jenssen remembers the doctors assuring her that, because the Duchenne mutation is linked to the ...

The FDA has granted Orphan Drug and Rare Pediatric Disease Designations to Somite’s cell replacement therapy for the ...

In 2023, two potential FDA approvals and three late-stage clinical trial readouts could shift the treatment landscape for Duchenne muscular dystrophy. The upcoming FDA approval decision for ...

Shares of Wave Life Sciences rose more than 10% in premarket trading Tuesday after the clinical-stage biotechnology company reported positive interim data from a mid-stage study of its WVE-N531 drug ...