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PPMD Provides $500,000 in Funding to Kinea Bio Through PPMD Venture Pathways Program to Support Next-Gen Midi-Dystrophin Gene Therapy Development
PPMD is excited to announce that the organization has provided $500,000 in funding to Kinea […]
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MDA advocacy program awards $140K to 7 rare disease groups
With projects spanning gene therapy research, patient advocacy, and air travel safety, seven U.S. organizations […]
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When I get overwhelmed by life as a caregiver, I count my joys
Pulling into a parking spot at my oldest son’s high school, I hit a parked […]
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Finding the Right One: Advice from Experts on Dating with a Disability
“So, are you dating anyone?” Could there possibly be five more scary words spoken to […]
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Update From PPMD’s 2024 PPMD Duchenne Healthcare Professionals Summit
PPMD recently hosted our fifth in-person Duchenne Healthcare Professionals Summit in Sanibel, FL. Over the […]
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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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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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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 […]
