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Watch: Dyne Therapeutics — Advancing DYNE-251, an Investigational Medicine for DMD (Webinar Recording)
Dyne Therapeutics recently joined PPMD for a community webinar to present initial clinical data from […]
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REGENXBIO Announces Completion of Enrollment in Cohort 2 and Additional Positive Interim Data in AFFINITY DUCHENNE® Trial
REGENXBIO Inc. has announced that enrollment has completed at dose level 2 of the Phase […]
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Natural history model estimates Duchenne MD trajectory
It may take an average of 11 years before people with Duchenne muscular dystrophy (DMD) […]
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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 […]
