– U.S. FDA has confirmed that screening and dosing may proceed in Study SRP-9005-101 for LGMD2C/R5 – Enrollment and dosing completed in Study SRP-9004-102 for LGMD2D/R3 – Data expected for SRP-9003 ...
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 ...
Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for ...
BridgeBio Pharma (NASDAQ:BBIO) shares rose after it announced positive results from its late-stage study of BBP-418 on patients with limb-girdle muscular dystrophy type 2I/R9. The study successfully ...
New York, Jan. 26, 2026 (GLOBE NEWSWIRE) -- In recognition of Rare Disease Day on February 28, the Muscular Dystrophy Association (MDA) is sharing powerful community stories throughout the month that ...
Muscular dystrophy (MD) is a group of genetic diseases that cause your muscles to progressively weaken and degenerate. There are several types of MD, each with its own symptoms, but they all involve ...
For more than 10 years, Napa in Newport has been the signature event to raise funds and awareness for CureDuchenne, a Newport ...
A retrospective cohort study found that, among 459 patients with DMD, constipation (32.5%) was the most common GI complication, followed by dysphagia (9.4%). Older age, lower body mass index, and use ...
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