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An emergency preparedness plan is crucial for those with disabilities
As Western Pennsylvania natives, my wife, Wendy, and I are both Steelers fans. Naturally, when the Steelers played the Dallas Cowboys a couple weeks ago, we tuned in. If you’re not a football fan, you’re most likely unaware that the game was delayed for about an hour and a half as a huge thunderstorm rolled…
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RNA-based approach shows promise for DM1 in cell models
Treatment with antimiRs, an RNA-based therapy approach, was able to correct molecular defects in a cell model of myotonic dystrophy type 1 (DM1) — a form of muscular dystrophy — according to the results of a new study. The researchers called their work “a significant step forward,” and noted that this strategy potentially “could be beneficial…
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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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