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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 […]
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Fear and pride as my son starts up with high school band
When I think about my son Charlie, I always remember my pregnancy with him. I’d […]
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US leadership emphasizes support for rare disease drug development
CureDuchenne is pleased that one of the main themes at the recent US FDA Cell […]
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Quest Podcast: Invisible People: Making the Rare Seen
In this Quest Podcast episode, we chat with a former pharmacist turned singer/songwriter who lives […]
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REGENXBIO Shares Positive Functional Data from Phase I/II AFFINITY DUCHENNE Trial of RGX-202
Today, REGENXBIO announced new data from its ongoing Phase I/II AFFINITY DUCHENNE® trial of RGX-202, […]
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PPMD’s Certified Care Center Program
PPMD continues to advocate to ensure families navigating a diagnosis of Duchenne and Becker muscular […]
