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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. […]
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Pepgen pulls plug on exon-skipping therapy for Duchenne MD
Following lackluster data from a Phase 2 clinical trial, Pepgen will discontinue developing PGN-ED051, its experimental […]
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Steroids and Stress Dosing: Understanding the Updated PJ Nicholoff Steroid Protocol
Last week, PPMD brought together leading experts to address steroids, stress dosing and the updated […]
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MDA Ambassador Guest Blog: How My MG Journey Has Shaped Who I Am Today
Courtney B. is a passionate advocate for living life to the fullest despite the challenges […]
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Why those of us with FSHD feel like we’re always in transition
During a recent facioscapulohumeral muscular dystrophy (FSHD) wellness group meeting via Zoom, we each shared […]
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PepGen ends development of Duchenne Exon-51 skipping program
PepGen has ended clinical trials of PGN-EDO51, their investigational therapy for Duchenne patients amenable to skipping exon […]
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A day of reflection leads me to a place of gratitude as a DMD mom
Once again, I’m writing from an uncomfortable chair in an infusion room as my son […]
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My Father’s Journey with ALS
Father’s Day can be a beautiful celebration — but also a tender time for many. […]
