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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…
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This time, new medical equipment is hard for me to accept
Our house is getting a new piece of medical equipment this week. That’s happened before, as we have three sons with Duchenne muscular dystrophy (DMD): Max, 18, Rowen, 15, and Charlie, 13. Over the past decade, in fact, our house has been a landing zone for lots of equipment as we’ve adapted to their disease…
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Rare pediatric disease designation granted to LAMA2-CMD therapy
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to MDL-101, an epigenetic editing therapy that Modalis Therapeutics is developing for LAMA2-related congenital muscular dystrophy (LAMA2-CMD) — a genetic condition that’s characterized by muscle wasting evident at birth or shortly thereafter. This FDA status recognizes MDL-101 for its potential to offer…
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WATCH: Interim Results from FORWARD-53 Study of WVE-N531 in Duchenne Muscular Dystrophy (Webinar Recording)
Wave Life Sciences recently joined PPMD for a presentation of interim results from the company’s ongoing Phase 2 FORWARD-53 study of WVE-N531 in individuals living with Duchenne muscular dystrophy who are amenable to exon 53 skipping, first released on September 24, 2024. Wave’s President and CEO and Chief Development Officer highlighted that WVE-N531 demonstrated substantial…
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DMD treatment WVE-N531 boosts dystrophin: Interim trial data
Treatment with exon 53-skipping therapy WVE-N531 led to significant increases in muscle dystrophin levels along with signs of improved muscle health and regeneration in boys with Duchenne muscular dystrophy (DMD). That’s according to new six-month interim data from the Phase 1b/2a FORWARD-53 trial (NCT04906460), which also found the treatment to be safe and well tolerated.…
