The U.S. Food and Drug Administration has granted orphan drug designation to Myonexus Therapeutics‘ MYO-102, a gene therapy for limb-girdle muscular dystrophy type 2D (LGMD 2D), the company announced. The FDA grants this designation to treatments with the potential to significantly improve the life of…
Emily Crossley was a well-known TV correspondent for Britain’s Channel 4, and a presenter for CNN International’s World Business Today program. But her own world was turned upside-down the day she learned her son, Eli, had Duchenne muscular dystrophy (DMD). “The initial diagnosis was so devastating. There’s no escape. It’s…
Throughout 2018, Muscular Dystrophy News Today provided daily coverage of new therapeutic strategies, clinical trials, and other topics related to muscular dystrophy (MD). As we look forward to reporting more news to patients, family members, and caregivers dealing with MD in 2019, here are the Top 10 most-read articles…
Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to treat rare diseases has never been greater. That’s the paradox faced by pharmaceutical companies as well as patient advocacy groups in…
Magnetic Resonance Imaging (MRI) can catch signs of damage to the heart in muscular dystrophy (MD) patients earlier than traditionally-used echocardiograms, according to a webinar providing updates on the Parent Project for Muscular Dystrophy (PPMD)’s cardiac initiative. The presentation took place Dec 12, which is almost halfway through…
Close to 85 percent of boys and young men with Duchenne muscular dystrophy have never seen a mental health professional, says a leading neurologist based in Washington, D.C. Mathula Thangarajh, MD, of Children’s National Medical Center, gave a talk, “Behavior and the Brain in Duchenne,” at the recent…
WVE-210201, an exon 51 skipping therapy, has shown positive safety and tolerability results in a Phase 1 clinical trial in boys with Duchenne muscular dystrophy (DMD), Wave Life Sciences announced. According to Wave Life Sciences, the positive safety data supports moving the therapy into a Phase 2/3 trial, planned to start…
At 21, Hawken Miller certainly doesn’t look like a typical Duchenne muscular dystrophy patient. For one thing, unlike most boys and young men with Duchenne, Hawken isn’t obese — or even overweight — even though he’s been on steroids for years. That’s because he eats a balanced, high-protein diet with generous…
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…
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