News

Until her son, Will, was diagnosed with Duchenne muscular dystrophy as a 7-year-old, speech pathologist Tiffany Cook had never heard of the disease. Today, it’s literally her full-time job. As family resource manager with the nonprofit organization CureDuchenne, Cook — who works from her home in Dallas — helps…

The University of Virginia (UVA) Children’s Hospital has been named a Certified Duchenne Care Center by Parent Project Muscular Dystrophy (PPMD), making it the 22nd such center recognized by the patient advocacy group for the quality and comprehensiveness of its neuromuscular program. UVA’s hospital is also the first CDCC with a prominent…

Hoping to make travel less difficult throughout England for disabled motorists, including those with muscular dystrophy, Muscular Dystrophy UK  and the government’s Department for Transport are working to bring accessible toilets to most highway service areas. Called Changing Places, the project is expected to be completed in the early…

For nearly half a year, Brenda Wong, MD, has led New England’s first and only interdisciplinary clinic devoted exclusively to boys and young men with Duchenne muscular dystrophy (DMD). The Duchenne Program at UMass Memorial Medical Center is located in Worcester, a 45-minute drive west of Boston. The clinic,…

Early safety and efficacy data from a Phase 1 trial suggest that Taiho Pharmaceutical’s investigational compound TAS-205 may hold the potential to treat patients with Duchenne’s muscular dystrophy (DMD). The results were reported in the study, “A phase I…

Like many mothers of boys with muscular dystrophy, Deborah Hall hasn’t had an easy life. Hall, the daughter of a chemical engineer and a stay-at-home mom, was one of six siblings growing up in Boaz, West Virginia. When her younger brother, John, was 3 years old, doctors diagnosed him with…

Body mass index (BMI) and blood pressure are modifiable factors that could help to slow heart deterioration in patients with Duchenne muscular dystrophy (DMD), according to a retrospective study. The study, “Increased Blood Pressure and Body Mass Index as Potential Modifiable Factors in The Progression of Myocardial Dysfunction…

Metformin, a diabetes treatment, eased disease progression and improved muscle function in female — and, to a lesser extent, in male — mice altered to have congenital muscular dystrophy with laminin α2 chain-deficiency (LAMA2-CMD), a study reports. Its researchers also reported that this type of congenital muscular dystrophy — the second…

Duchenne UK announced that it has awarded a sizable grant to Evox Therapeutics to advance in early testing a way of using gene therapy to deliver — without a virus as a transport agent — the dystrophin gene or shorter versions of it to people with Duchenne muscular dystrophy. The group’s…

This month’s Democratic takeover of the U.S. House of Representatives following the Nov. 6 midterm elections could be good news for Americans with rare diseases, says the National Organization for Rare Disorders (NORD), which represents 280 patient advocacy organizations. “The entire community advocated against the repeal of the Affordable Care…