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Join Us for the Fall PPMD Lighthouse Workshops!

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CDCC Spotlight: Helen DeVos Children’s Hospital

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Sam’s Night Has Another Record-Breaking Year, Raising Over $1,400,000 for PPMD

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WATCH: Supporting Families Through Decision Making (Webinar Recording)

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WATCH: Affinity Duchenne™ and Affinity Beyond™: Updates on Investigational RGX-202 from REGENXBIO (Webinar Recording)

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FibroGen announces discontinuation of LELANTOS-2 Open Label Extension

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Santhera Receives Positive European Medicines Agency (EMA) CHMP Opinion Recommending Approval of Vamorolone in European Union

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Finding and Accepting Help

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What is Palliative Care? Understanding an additional layer of support

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REGENXBIO announces interim clinical data from Phase I/II AFFINITY DUCHENNE™ trial of RGX-202

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