Pfizer has announced a delay in opening U.S. sites for its Phase 3 clinical trial CIFFREO, evaluating the investigational gene therapy PF-06939926 in boys with Duchenne muscular dystrophy (DMD). The delay is caused by questions…
Sarepta Therapeutics has announced positive results from a global study evaluating its investigational treatment SRP-5051 in people with Duchenne muscular dystrophy (DMD)Â who are amenable to exon 51 skipping. Results from part A of the…
Ray Jordan and Cyndi Segroves, a married couple with facioscapulohumeral muscular dystrophy (FSHD), haven’t let their disability get in the way of anything. They’ve traveled from their Arizona home to destinations around the world — from Hawaii and New…
The glint of armor demands attention. The jousting lance was slung to the side of a great black horse. The rider, shield in hand, mounted the stallion and paraded about a wooden bleacher. The horse coolly pulled one leg up, then the other, muscles rolling with each movement.
The muscles of people with Duchenne muscular dystrophy (DMD) show profound impairment, with changes in fatty molecules and energy metabolism, as compared with those of their healthy peers, a small French study revealed. While previous studies have reported such metabolic changes in DMD patients, this was the first…
When Alfie was first diagnosed, I went searching. I needed answers. I needed to know that my little boy would be OK. I searched every website and every social media platform. Some days I got so obsessed that I’d have to force myself to stop.
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read the latest stories from the conference. People with myotonic dystrophy type 2 (DM2) tend to be older at symptom onset than those with myotonic…
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read the latest stories from the conference. Women with limb girdle muscular dystrophy type R9 (LGMDR9) are not at a greater risk of pregnancy complications,…
More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read the latest stories from the conference. Once-weekly corticosteroid treatment was safe and well-tolerated in people with Becker muscular dystrophy (BMD) or limb-girdle muscular dystrophy…
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