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Fun summer activities that are still doable with Duchenne
I often read social media posts from moms asking for safe and fun ideas for their children to enjoy during summer break. When my boys were younger, I worried about that as well. I wanted my three sons with Duchenne muscular dystrophy (DMD) — Max, 18, Rowen, 15, and Charlie, 13 — to have the…
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Losmapimod improved, stabilized FSHD upper limb function: Study
One year of losmapimod taken twice a day improved or stabilized upper limb function and muscle strength in adults with facioscapulohumeral muscular dystrophy (FSHD), according to an open-label pilot study. Blood and muscle tests also showed the therapy candidate successfully engaged with its intended target and had a favorable safety and tolerability profile. Details of…
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NHL All-Star Ryan Getzlaf Returns to the Links for the 13th Annual Getzlaf Golf Shootout on September 13-14, Benefiting CureDuchenne
Event Has Raised More Than $5.8 Million Over the Last 12 Years to Drive a Cure for Duchenne Muscular Dystrophy NEWPORT BEACH, Calif., (July 12, 2024) — CureDuchenne, a leading global nonprofit focused on finding and funding a cure for Duchenne muscular dystrophy, will partner once again with NHL All-Star and retired Anaheim Ducks captain…
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Ullrich Awareness Day
Finally, after years of battling and campainging, we now have an official Ullrich Muscular Dystrophy Awareness Day! It’s a particularly rare form of muscular dystrophy, which you probably haven’t heard of – until now. This awareness day means a great deal to me, since I was born with Ullrich congenital muscular dystrophy 35 years ago.…
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Protected: FDA Approval of ELEVIDYS: Key Takeaways from the CureDuchenne Webinar
This content is password protected. To view it please enter your password below: Password: The post Protected: FDA Approval of ELEVIDYS: Key Takeaways from the CureDuchenne Webinar appeared first on CureDuchenne.
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What I want people to know about FSHD and its progression
As I was finishing up a recent interview with the host of “FSHD Radio: Straight Talk with Tim Hollenback,” a service of the FSHD Society, Tim’s final question was, “If there’s one thing you’d like people to know about facioscapulohumeral muscular dystrophy [FSHD], what would it be?” It was a great question. I’m not sure…
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Twins offer insights into DM1 cognitive deficit contributors
A pair of identical twins with myotonic dystrophy type 1 (DM1) were found to have different deficits in social cognition that corresponded with differing patterns of brain tissue loss on MRI scans, a case report shows. Researchers believe the findings highlight that, while genetic factors can contribute to brain development, differences in lifestyle and environment can…
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PPMD 2024: Gene therapy trials recruiting patients around the world
With the first gene therapy for Duchenne muscular dystrophy (DMD) now approved in the U.S. for most people with the disease, clinical trials are underway to better understand the safety and efficacy of the approved treatment, find strategies to expand its use, and develop the next generation of DMD gene therapies. The current and evolving…
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In the dance of who does what with Duchenne, it’s OK to ask for help
“What can I do to help?” It was my husband’s answer to the long rant I’d just finished. I didn’t know I needed an answer, but his caught my attention. Beyond the weight of my responsibilities, I was trying to carry myself — and the heft of that was crushing me. Of my sons, three…
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PPMD 2024: Trials due for DMD gene therapies, cell regeneration
In its 30-year history, Parent Project Muscular Dystrophy (PPMD) has helped fund the development of many experimental treatments for Duchenne muscular dystrophy (DMD) and some of these are about to be tested in clinical trials. Several of these up and coming treatments, including strategies for gene therapy and approaches to regenerate muscle cells, were showcased…
