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DYNE-101 is leading to functional gains for DM1 patients in trial
The investigational therapy DYNE-101 worked as expected and led to functional improvements for people with […]
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PPMD Advocacy in the 119th Congress and the 47th Administration
As the 119th Congress and the 47th Administration prepare to take office, PPMD is committed […]
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PPMD Hosts 2025 Duchenne Healthcare Professionals Summit in San Diego, California
PPMD is excited to host the sixth in-person Duchenne Healthcare Professionals Summit in San Diego, […]
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GIVI-MPC named FDA orphan drug for treatment of Becker MD
The U.S. Food and Drug Administration (FDA) has granted IPS Heart’s GIVI-MPC — a stem cell […]
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How my FSHD progression leads to out-of-body experiences
Many times over the years since the 1989 car crash that nearly killed me, I’ve […]
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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 […]
