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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
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FDA grants orphan drug status to ISX9-CPC for heart issues in DMD
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to ISX9-CPC, IPS
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Regenxbio reports positive interim data from Phase 1/2 microdystrophin gene therapy trial
Regenxbio shared positive data from the AFFINITY DUCHENNE trial of RGX-202. In addition to robust
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Building empathy for Duchenne experiences through theater
Last Thursday, I participated in a workshop that used experiential theater to examine healthcare conversations.
