– 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 ...
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 ...
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 ...
"My medicine is making me stronger," 7-year-old Hudson Sanford says after receiving a breakthrough gene therapy for Duchenne Muscular Dystrophy Cara Lynn Shultz is a writer-reporter at PEOPLE. Her ...
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 ...
For more than 10 years, Napa in Newport has been the signature event to raise funds and awareness for CureDuchenne, a Newport ...
Your muscles need continuous maintenance to stay strong and healthy. Duchenne muscular dystrophy (DMD) happens when there is a change in a person's genetic instructions that affects the production of ...
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