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PPMD’s 30th Annual Conference Session Recordings

Thank you to everyone who helped us celebrate three decades of progress, community, and resilience at PPMD’s 30th Annual Conference, whether you joined us in person in Orlando, or tuned in virtually! PPMD’s 30th Annual…

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PPMD Applauds FDA Launch of Rare Disease Innovation Hub — A Milestone for Rare Disease Patients

Parent Project Muscular Dystrophy (PPMD) welcomes the recent announcement by the United States Food and Drug Administration (FDA) of the “Rare Disease Innovation Hub”. This initiative marks a pivotal moment in the journey towards better…

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Capricor Therapeutics Announces Positive Skeletal Muscle and Cardiac Function Data from HOPE-2 OLE Study Data of Deramiocel (CAP-1002)

Capricor Therapeutics today announced additional positive 3-year safety and efficacy results from the company’s ongoing HOPE-2 open label extension (OLE) study of deramiocel (CAP-1002) for the treatment of Duchenne. CAP-1002 is the company’s novel cell…

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PPMD Convenes Leading Experts to Discuss Topics in Becker Care Ahead of 30th Annual Conference

Leading into PPMD’s 30th Annual Conference, PPMD is excited to host a professional meeting focusing on Becker muscular dystrophy care. Over the course of two days, attendees from across subspecialties, academic medical centers, and industry…

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REGENXBIO Announces Expansion of AFFINITY DUCHENNE® Trial to Include New Cohort of Younger Patients

REGENXBIO has announced it initiated enrollment in a new cohort of patients ages 1-3 in its Phase I/II AFFINITY DUCHENNE® trial. The trial is evaluating the safety and efficacy of RGX-202, an investigational gene therapy…

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FDA Expands ELEVIDYS Label Indication for Ages 4 Years and Older

PPMD is excited to share that the FDA has made the decision to expand the labeled indication for ELEVIDYS, a micro-dystrophin gene therapy used to treat people with Duchenne muscular dystrophy, to include individuals at…

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PAAC Travel Tips

Anyone living with Duchenne muscular dystrophy knows that there are extra steps to EVERYTHING. Traveling with medical equipment requires planning and organization. PPMD’s Adult Advisory Committee (PAAC) has put together several resources with tips that…

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Newborn Screening for Duchenne: PPMD’s Collaborative Future Planning Efforts

On Saturday, June 8th, PPMD convened a vital meeting of physicians, physical therapists, genetic counselors, data scientists, and other key opinion leaders to discuss the development of data infrastructure and care planning for newborns that…

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Cranbury Pharmaceuticals (Tris Pharma) Receives FDA Approval for First Generic Version of Emflaza® (deflazacort) for Duchenne

Cranbury Pharmaceuticals, a subsidiary of Tris Pharma, today announced the U.S. Food and Drug Administration (FDA) approved the Abbreviated New Drug Application (ANDA) for the first generic version of Emflaza® oral suspension (deflazacort) for the…

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Build Your Own Care Binder at PPMD’s 30th Annual Conference

PPMD is excited to share that at our 30th Annual Conference, taking place June 27-29, 2024, in Orlando, Florida, the PPMD Care team will host a “DIY Care Binder” station where families can create a…

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