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Why I’m looking forward to celebrating my first Valentine’s Day
Amid a topsy-turvy start to 2024, I’ve marked a date on my calendar I’ve been
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REGENXBIO Announces Completion of Enrollment in Cohort 2 and Additional Positive Interim Data in AFFINITY DUCHENNE® Trial
RegenXBio shared positive updates from their Phase 1/2 AAV micro-dystrophin gene therapy clinical trial for
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How I realized I was doing too much for my sons with DMD
I do too much. You might assume I’m talking about caregiving because you’re reading a
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European committee not in favor of renewing Translarna’s approval
A committee of the European Medicines Agency (EMA) has maintained its initial recommendation not to
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Duchenne Newborn Screening Bill Passes Arizona Senate
Earlier this week, Duchenne parent and PPMD Advocate Jill Castle testified before the Arizona State
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I welcome Agamree’s approval with renewed optimism
I received an email on Jan. 12 from Duchenne UK, an important Duchenne muscular dystrophy
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Keep Dreaming for the Future – Even with DMD
By Jacob Gapko Jacob Gapko is 46 years old and has Duchenne muscular dystrophy. He uses
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Watch: Navigating Steroid Choices – Understanding the Process, Pathways, and Policies (Webinar Recording)
Last week, PPMD was joined by Kathy Mathews, MD (Neurologist), Christina Trout, RN, MSN (Clinic
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‘Favorable risk-benefit profile’ seen for DMD treatment SRP-5051: Trial
SRP-5051 is able to increase the production of dystrophin protein in people with Duchenne muscular
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Sarepta Therapeutics Announces Positive Data for Duchenne Muscular Dystrophy Amenable to Skipping Exon 51
Sarepta Therapeutics reported positive data for SRP-5051, their next-generation exon skipping agent designed for individuals amenable
