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My original diagnosis was incorrect. Here’s why that’s important.
In August 1985, I was diagnosed with Becker muscular dystrophy. At that time, there was […]
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Dyne plans to submit for FDA Accelerated Approval for Duchenne exon 51 skipping in 2026; Trial still recruiting participants.
Dyne Therapeutics, which received early funding from CureDuchenne, plans to pursue US Accelerated Approval from […]
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Waking up on Christmas Eve made me scared and anxious
The Christmas decorations are down and tucked into storage for another year. The house is […]
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Introducing PPMD’s Dystrophinopathy Clinical Research Network (DCRN): Advancing the Research Evolution
In our rapidly evolving therapeutic landscape, there is a critical need for cohesive infrastructure that […]
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Avidity Biosciences Announces Plans to File Biologics License Application for Delpacibart Zotadirsen (AOC 1044)
Avidity Biosciences, Inc. has announced its plans for filing a Biologics License Application (BLA) with […]
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Gene-editing therapy for LGMD shows promise in mouse model
A gene-editing therapy designed to correct a defect in the DYSF gene — one that’s […]
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PPMD’s Race to End Duchenne Program Celebrates 20 Years of Going the Distance to End Duchenne
PPMD is celebrating the 20th anniversary of our endurance fundraising program, Race to End Duchenne, […]
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How ‘The Remarkable Life of Ibelin’ moved me as a DMD survivor
A few days ago, I watched the Netflix documentary “The Remarkable Life of Ibelin.” I […]
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Application finalized for DMD cell therapy CAP-1002, now deramiocel
Capricor Therapeutics has completed its submission of a biologics license application (BLA) seeking U.S. approval […]
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Thank You for Joining us to Advance the Research Evolution
As we enter PPMD’s fourth decade and a new era in Duchenne research, I am […]
