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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…
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PPMD 2024: 4 therapies aiming to slow DMD progression now in trials
Four experimental treatments — deramiocel, sevasemten, satralizumab, and tadalafil — all in clinical testing and all aiming to slow the progression of Duchenne muscular dystrophy (DMD), were spotlighted in a session at last week’s Parent Project Muscular Dystrophy (PPMD) conference. In Duchenne MD, mutations lead to a lack of functional dystrophin, a protein that normally…
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A rough month makes me grateful for what I’ve got
Last month was tough for me. Although celebrating my partner’s birthday on June 12 was a welcome distraction, I still had a June I’d like to forget, mainly because I spent the entire month recovering from a foot infection that hospitalized me for the last three days of May. And after that I had an…
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FDA APPROVAL OF ELEVIDYS
This webinar discusses a groundbreaking gene therapy treatment for Duchenne Muscular Dystrophy (DMD), focusing on a method to introduce a miniaturized version of the dystrophin gene using a viral vector. This therapy, while not a cure, aims to improve the quality of life for patients. The treatment involves a single dose, but patients must undergo…
