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PepGen ends development of Duchenne Exon-51 skipping program
PepGen has ended clinical trials of PGN-EDO51, their investigational therapy for Duchenne patients amenable to skipping exon 51, as PGN-EDO51 failed to achieve target dystrophin levels in their CONNECT1 trial. The company intends to wind down all DMD-related research and development activities. While it is a disappointing outcome, we’d like to thank PepGen, who CureDuchenne provided early funding to,…
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My Father’s Journey with ALS
Father’s Day can be a beautiful celebration — but also a tender time for many. For those whose fathers are no longer with us, this day may carry sorrow along with sweet memories. At Muscular Dystrophy Association (MDA), we support the community with those feeling that loss, and offer our empathy, love, and resources. This…
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A day of reflection leads me to a place of gratitude as a DMD mom
Once again, I’m writing from an uncomfortable chair in an infusion room as my son Max participates in a clinical trial. I frequently visit this room with my three sons — Max, 19; Rowen, 16; and Charlie, 14 — who live with Duchenne muscular dystrophy (DMD). It’s a boring day. Well, let me take that…
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Accessible Air Travel Is Ready for Takeoff. Can It Avoid Turbulence?
The past year featured the most disability-positive changes for air travel in decades. While there is still a way to go before air travel is fully accessible, policy and rule changes promise to make it safer and more dignified for people with disabilities. Shannon Wood, MDA’s Director of Disability Policy Most of the changes came…
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Simply Stated: Updates in Ryanodine Receptor 1-Related Disorders (RYR-1-RD)
RYR-1-Related Disorders (RYR-1-RD) are a group of rare, inherited muscle disorders caused by variants in the RYR1 gene. This gene encodes the ryanodine receptor type-1 (RyR1) protein, which is important for muscle function and the ability of muscles to contract. People with RYR-1-RD experience muscle weakness, and may exhibit a variety of other symptoms, including a…
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CureDuchenne invests in new redosable DMD gene therapy
CureDuchenne is investing $1 million in Entos Pharmaceuticals to support the development of a new gene therapy for Duchenne muscular dystrophy (DMD) that aims to overcome the limitations of existing gene therapies. “This investment underscores our continued use of venture philanthropy to catalyze progress [toward] transformative treatments for Duchenne,” Debra Miller, founder and CEO of…
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PepGen Announces Discontinuation of PGN-EDO51 Development
We are disappointed to share that PepGen Inc. announced the company’s decision to end development of PGN-EDO51, its investigational therapy for the treatment of people with Duchenne amenable to exon 51 skipping. PepGen indicated that the CONNECT1-EDO51 trial will stop dosing, and the CONNECT2-EDO51 did not dose any participants and will now be closed. Clinical…
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DMD treatment SAT-3247 safe in trial, shows early signs of efficacy
Satellos Bioscience’s SAT-3247 was safe, well-tolerated, and showed initial signs of increasing muscle strength in five adults with Duchenne muscular dystrophy (DMD), the company said. “We believe the findings from this Phase 1b study support our plan to advance SAT-3247 into a placebo-controlled Phase 2 trial,” Satellos CEO Frank Gleeson said in a company press…
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Governor Abbott Signs Duchenne Newborn Screening into Law in Texas
We are pleased to share a significant milestone for the Duchenne community: Texas Governor Greg Abbott has officially signed Senate Bill 1044 into law! This legislation requires the Texas Department of State Health Services to add Duchenne to the state’s newborn screening panel, a major advancement in our fight to ensure earlier diagnosis and intervention…
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Speech Devices Improve Communication When ALS Affects the Voice
As amyotrophic lateral sclerosis (ALS) progresses, it affects many aspects of daily life. One of the most significant impacts is its effect on speech. “Communication is such a key factor in who we are as people and how we represent and express ourselves that this is one of the more devastating aspects of the disease…
