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Avidity’s del-desiran for DM1 named orphan drug in Japan
The Japan Ministry of Health, Labour and Welfare (MHLW) has granted orphan drug status to […]
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Travelers with Disabilities Share Their Top Tips to Avoid Mishaps
Travel blogger Cory Lee lives with spinal muscular atrophy (SMA) and travels with his 400-pound […]
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Setback in gene therapy for Duchenne muscular dystrophy as immune system emerges as key barrier
A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, […]
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Congress Moves Forward on Medicaid Cuts: What You Need to Know
This week, we received concerning news from Washington: Congress has passed a budget resolution that […]
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Vamorolone up for approval in Canada as treatment for DMD
Health Canada has agreed to review an application by Kye Pharmaceuticals seeking the approval of […]
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Watch: Wave Life Sciences – Update on FORWARD-53 Study of WVE-N531 (Webinar Recording)
Wave Life Sciences recently joined PPMD for a community webinar to provide an update on […]
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MDA Ambassador Guest Blog: Spreading Awareness One T-shirt at a Time
Dwayne Wilson is 57 years old. He is originally from Southern California and lived in […]
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A Singaporean theater production reveals caregiving complexity
I recently had the opportunity to attend a Singaporean theater production titled “Supervision,” thanks to […]
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Independent committee favors continuing Elevidys dosing in trials
An independent data monitoring committee (DMC) favors continuing dosing Elevidys (delandistrogene moxeparvovec-rokl) to people with […]
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In Case You Missed It…
Quest Media is an innovative adaptive lifestyle platform from MDA. With the power of this […]
