News

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…

The ongoing Phase 2 HOPE-2 study has been cleared to continue exploring the potential of CAP-1002 therapy in boys and young men with advanced Duchenne muscular dystrophy (DMD). An independent Data and Safety Monitoring Board (DSMB) made the decision after its thorough safety and futility review and the…

Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state today tests for all 35 disorders recommended under a federal screening panel, and even in those that come close, rare…