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Sarepta Announces Discontinuation of SRP-5051 Development for Duchenne
Sarepta Therapeutics today announced that the company is discontinuing its SRP-5051 (vesleteplirsen) development program, including […]
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WATCH: Exploring Muscle Regeneration – A New Approach with Satellos (Webinar Recording)
Satellos recently joined PPMD for a community webinar exploring a new approach for the treatment […]
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Phase 1/2 trial of del-brax enrolling new, possibly pivotal FSHD group
A new patient group is being enrolled in a Phase 1/2 trial of delpacibart braxlosiran […]
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As a Duchenne mom, I know I can do hard things
The seasons are changing in central Nebraska. The leaves have turned yellow and orange, a […]
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CureDuchenne partners on World of Warcraft research fundraiser
CureDuchenne is partnering with Blizzard Entertainment, maker of the massively multiplayer online role-playing game World […]
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My trip to the Idlewild amusement park was wild, but worth it
Idlewild, which is near Ligonier, Pennsylvania, is an old-style amusement park that’s been around since […]
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FDA Accepts Translarna NDA Resubmission for Review
A Letter From PPMD’s Pat Furlong I am thrilled to share that the FDA has […]
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Flu Season Ready: Protecting Yourself and Your Family
With the start of school and the onset of winter, we can expect a rise […]
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FDA awards LAMA2-CMD therapy MDL-101 orphan drug status
MDL-101, Modalis Therapeutics’ epigenetic editing therapy for LAMA2-related congenital muscular dystrophy (LAMA2-CMD), has been granted […]
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Duchenne affects my entire life, down to a DIY project
I’m a mom to seven children: Lexi, 23; Max, 18; Chance, 17; Rowen, 15; Charlie, […]
