Fulcrum Therapeutics announced it has launched ReDUX4, a Phase 2b clinical trial designed to assess the safety and efficacy of losmapimod, an investigational treatment for facioscapulohumeral muscular dystrophy (FSHD) in patients with a genetically confirmed diagnosis of FSHD. The Phase 2b ReDUX4 trial (NCT04003974) is recruiting…
News
Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people who’ve made it to adulthood. Neena Nizar doesn’t have to imagine. The 41-year-old English professor at Metro Community College in Elkhorn,…
An adverse event report was erroneously submitted for a patient enrolled in Study-102 testing Sarepta Therapeutics’ micro-dystrophin gene therapy, an experimental treatment for Duchenne muscular dystrophy (DMD), the company announced. The board in charge of the study’s safety found no reason to stop the trial. According to…
Jiffy Lube has launched its 8th annual Muscle Up! campaign, which supports the Muscular Dystrophy Association’s (MDA) Summer Camp program as well as research and family services. Throughout August, Jiffy Lube patrons at participating service centers nationwide are encouraged to donate at least $3 to…
The Children’s Hospital of Richmond (CHoR), Virginia, has been named Parent Project Muscular Dystrophy’s (PPMD) 26th Certified Duchenne Care Center (CDCC). PPMD’s program, established in 2014, recognizes facilities with outstanding neuromuscular programs providing comprehensive care with recognized standards to all living with Duchenne muscular dystrophy (DMD), a multi-systemic…
On a recent summer morning off the coast of Bat Yam, south of Tel Aviv, a dozen Israeli volunteers in wetsuits splashed in the sparkling blue waters of the Mediterranean Sea. But they weren’t there just for fun. The focus of their attention: three young boys gripping specially modified surfboards…
Move over, Sarepta Therapeutics, Solid Biosciences, PTC Therapeutics and Pfizer. Vertex Pharmaceuticals, which dominates the cystic fibrosis drug market, has just entered the multibillion-dollar race to find effective treatments — and maybe even cures — for two other rare diseases: Duchenne muscular dystrophy…
Aiming to enhance patients’ lives, a team of University of Manitoba biomedical students has produced autonomous designs — including a wheelchair hand warmer for those living with muscular dystrophy (MD). The overarching goal during the University of Manitoba Biomedical Engineering Design (BMED) Team’s first year was…
Years ago, doctors believed that no exercise was best for boys with Duchenne muscular dystrophy (DMD). Today, DMD experts all agree: exercise extends lives. But what kind of exercise is ideal for Duchenne kids? How much is too much? And what about stretching, night splints and specialized equipment? Physical…
Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of teen births per 100,000 teenage girls, and has the nation’s third-highest rate of uninsured residents — with 13.9% of all Oklahomans lacking health coverage. As if…
Recent Posts
- MDA 2026: This year’s MDA meeting provided a glimpse into the future
- I’m thankful for a big family that gives my DMD sons a full social life
- MDA 2026: Dyne advances DMD therapy z-rostudirsen toward approval
- 17-year-old advocate shares her NMOSD story to raise awareness
- MDA 2026: ‘Body of evidence for Elevidys continues to grow’