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PPMD Announces Children’s Hospital of Philadelphia as CDCC, Recipient of Inaugural Clinical Research Designation
PPMD is pleased to announce the expansion of its renowned Certified Duchenne Care Center (CDCC) […]
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Duvyzat conditionally approved by EC to treat Duchenne MD
The European Comission has conditionally approved Duvyzat (givinostat) to be taken alongside corticosteroids for treating […]
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Capricor’s Advisory Committee meeting with FDA to be held July 30, 2025
Capricor Therapeutics, which received early funding from CureDuchenne, announced regulatory updates for their Deramiocel program […]
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After incredible hardships, I still feel blessed
I’m between two anniversaries. Today is the 36th anniversary of the collision I had with […]
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Viral vector in LGMD2E gene therapy gets new FDA designation
The U.S. Food and Drug Administration (FDA) granted platform technology designation to the viral vector […]
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The Joys (and Lessons) of Fatherhood from MDA Ambassadors
As every father knows, parenthood is an incredible journey filled with joy, challenges, victories, growth, […]
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Inclusive Summer Activities
As the school year comes to a close, we are all looking forward to a […]
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Watch: Dyne Therapeutics — The Clinical Impact of the FORCE™ Platform for Duchenne (Webinar Recording)
Dyne Therapeutics recently joined PPMD for a community webinar on the DELIVER clinical trial evaluating […]
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We’re our own best experts, but what if we’re incapacitated?
I believe that many adults living with rare diseases can and do become experts in […]
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In Case You Missed It…
Quest Media is an innovative adaptive lifestyle platform from MDA. With the power of this […]
