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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 […]
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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. […]
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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
