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Gene therapy at low dose helps two OPMD patients with swallowing
A single low dose of the investigational gene therapy BB-301 helped with swallowing for the first two people with oculopharyngeal muscular dystrophy (OPMD) treated in a Phase 1b/2a clinical trial, according to an update from the therapy’s developer, Benitec Biopharma. Trial findings to date, which include reasonable treatment safety being seen, were presented at the…
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Catch Up on World Muscle Society 2024
Each year, the World Muscle Society (WMS) Annual Congress brings together the best minds in neuromuscular research, clinical care, patient advocacy, and industry. It offers a dynamic platform for sharing new data, fostering collaborations, and advancing the science behind therapies for diseases like Duchenne muscular dystrophy. This year’s event in Prague, Czech Republic, featured talks…
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WATCH: SHIELD DMD – A Bone Health Study in Duchenne (Webinar Recording)
Bone health is an important aspect of living with Duchenne muscular dystrophy. Roche recently joined PPMD for a webinar during which Maitea Guridi, a Principal Clinical Scientist at Roche, shared learnings around the natural history of bone health in Duchenne. We discussed why fractures occur, with Maitea explaining that communication between muscles and bone is…
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Capricor now seeking FDA approval of DMD therapy deramiocel
Capricor Therapeutics has started its rolling submission seeking U.S. approval of deramiocel, a cell therapy candidate to treat cardiomyopathy, a disease of the heart muscle, in people with Duchenne muscular dystrophy (DMD), the company announced. Rolling submission means that Capricor will be submitting parts of the process to the U.S. Food and Drug Administration (FDA) as…
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Every week, we try to organize chaos in our family of 9
What’s a typical week as a caregiver of three sons with Duchenne muscular dystrophy (DMD) like? As a primary caregiver to Max, 18, Rowen, 15, and Charlie, 13, my honest answer is that I wish I knew! There is no typical week. I also have four children who don’t have DMD: Lexi, 23, Chance, 17,…
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Capricor Initiates Rolling Submission of Biologics License Application (BLA) for Deramiocel (CAP-1002)
Capricor Therapeutics Initiates Rolling Submission of Biologics License Application (BLA) for Deramiocel (CAP-1002) for the Treatment of Duchenne Cardiomyopathy Capricor Therapeutics announced it has begun the rolling submission of its Biologics License Application (BLA) with the FDA seeking full approval for deramiocel (CAP-1002) for the treatment of Duchenne-cardiomyopathy. This submission process is expected to be…
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13th Annual Getzlaf Golf Shootout Raises Nearly $500,000 for CureDuchenne, Welcoming Troy and Danielle Terry as New Hosts
Ryan and Paige Getzlaf Pass the Torch to Anaheim Ducks All-Star Troy Terry and His Wife Danielle, Ushering in a New Era for the Iconic Event NEWPORT BEACH, Calif., (September 20, 2024) – The 13th annual Getzlaf Golf Shootout, a marquee event supporting CureDuchenne’s mission to find a cure for Duchenne muscular dystrophy, raised nearly…
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Protected: 2024 Annual Congress of the World Muscle Society Updates
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Educating all ages about living with muscular dystrophy
In my four decades of life with limb-girdle muscular dystrophy, I’ve become comfortable educating everyone I meet about my quality of life and the ways all of us living with chronic, rare conditions have been given a wonderful chance to influence societal views of the disabled community. I’ve found it most effective to keep my…
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C-Path to lead new task force seeking to advance LGMD therapies
The Critical Path Institute (C-Path) is launching a task force to advance the development of therapies for limb-girdle muscular dystrophy (LGMD) and will lead the efforts of its members to find a new treatment to, among other goals, slow or stop the progression of the muscle-wasting disease. Formed under the nonprofit’s Rare Disease Cures Accelerator-Data…
