Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for ...

Endoxifen program into rare pediatric neuromuscular disease along with previously received Rare Pediatric Disease Designation ...

Zeleciment basivarsen (z-basivarsen) demonstrated sustained functional improvement across multiple clinical measures in the ongoing ACHIEVE ...

Muscular dystrophy, known as DMD, is a rare and fatal genetic disease that primarily affects boys, slowly robbing them of their ability to walk, breathe and live independently. Until now, there have ...

Commercial launch in Switzerland anticipated in H2 2026Pratteln, Switzerland, January 15, 2026 – Santhera Pharmaceuticals (SIX: SANN) announces ...

Solid Biosciences Inc. company and executive profile by Barron's. View the latest SLDB company infomation and executive bios.

Minnesota (WCCO) -- An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment. Colton Belluzzo was diagnosed with a form of muscular ...

Editor’s note: This is an automatically generated transcript. Please notify editor@healio.com if there are concerns regarding accuracy of the transcription. Muscular dystrophy, as a category, is quite ...

Panelists discuss how exciting ongoing research efforts are expanding similar gene therapy technologies to other muscular dystrophies like FSHD and myotonic dystrophy, using strategies to knock down ...

Edgewise Therapeutics Inc. (NASDAQ:EWTX) is one of the best up and coming stocks to invest in now. On June 26, Edgewise Therapeutics announced positive results from its sevasemten program for Becker ...