Endoxifen program into rare pediatric neuromuscular disease along with previously received Rare Pediatric Disease Designation ...
The Star Press on MSN
Accessible van giveaway helps Hoosiers like Kim Ball
Kim Ball received an accessible van from the Muscular Dystrophy Family Foundation. Applications for the next giveaway are ...
Parent Project Muscular Dystrophy (PPMD), the largest U.S. non-profit leading Duchenne and Becker muscular dystrophy care, research, and advocacy efforts, celebrated a powerful and inspiring Walt ...
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...
Myotonic dystrophy type 1 (DM1) is the most common form of adult-onset muscular dystrophy, affecting about 1 in 8,000 people. While it is well known for causing muscle weakness and stiffness, DM1 also ...
A gene therapy for Duchenne muscular dystrophy caused complications to the liver, prompting a review of its use for younger patients. By Christina Jewett The Food and Drug Administration said on ...
Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...
Almost a year after buying Kate Therapeutics, Novartis has scooped up another San Diego-based muscle dystrophy biotech in one of the biggest acquisitions of 2025 so far. The Swiss pharma is paying $12 ...
Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...
Sarepta Therapeutics Inc. (NASDAQ:SRPT) stock is trading higher on Wednesday, with apparently no news to justify the movement. This week, Sarepta will present data from its gene therapy, ...
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