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Summer fun

Summer Fun: Accessible Activities for All Ages and Interests

As the school year wraps up, we are all looking forward to a fun-filled summer. It can be overwhelming to plan outings and activities considering accessibility and necessary accommodations to keep everyone safe. Along…

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Capricor Therapeutics Announces Positive 3-Year Efficacy Results from HOPE-2 Open Label Extension Study of CAP-1002

Capricor Therapeutics has announced positive 3-year results from the ongoing HOPE-2 open-label extension (OLE) study of CAP-1002 for the treatment of Duchenne. CAP-1002 is the company’s novel cell therapy. The HOPE-2 is a double-blind, randomized,…

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NS Pharma Shares Update on VILTEPSO® (Viltolarsen) Phase 3 Study

NS Pharma, Inc. has shared preliminary analysis results from RACER53, the global Phase 3 clinical trial of NS-065/NCNP-01 (viltolarsen). Viltolarsen is an antisense oligonucleotide drug intended to treat patients with Duchenne who are amenable to…

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Dyne Therapeutics Shares New Clinical Data from DELIVER Trial of DYNE-251 in Duchenne Patients

Dyne Therapeutics has shared new clinical data from the ongoing DELIVER trial of DYNE-251 in patients with Duchenne who are amenable to exon 51 skipping. DYNE-251 is an exon skipping product that combines a PMO…

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Study to Explore Barriers to Diverse Clinical Trial Participation in Duchenne Published

PPMD is pleased to share that the article, “Barriers to diverse clinical trial participation in Duchenne muscular dystrophy: Engaging Hispanic/Latina caregivers and health professionals,” has been published in the Orphanet Journal of Rare Diseases. This…

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PTC Therapeutics Announces European Commission Returns Translarna™ Opinion to CHMP For Re-evaluation

PTC Therapeutics, Inc. announced today that the European Commission (EC) has decided not to adopt the Committee for Medicinal Products for Human Use’s (CHMP) January 2024 negative opinion on the annual renewal of the conditional…

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Congress Passes FAA Reauthorization Featuring Monumental Accessibility Improvements

This week marked a significant milestone as Congress successfully passed the bipartisan Federal Aviation Administration (FAA) reauthorization, officially known as HR 3935, the Securing Growth and Robust Leadership in American Aviation Act. This legislation, now…

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Mapping Out a Path Forward: PPMD Convenes Representatives from 21 Academic Centers and 11 Industry Partners for 2024 Cardiac Care Workshop

Last week, PPMD held the 2024 Cardiac Care Workshop in Baltimore, Maryland, which included 40 academics and professionals and representatives from 11 industry partners to discuss how to move beyond the current state of cardiac…

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PPMD’s Pat Furlong Honored with ASGCT’s Sonia Skarlatos Public Service Award

PPMD is proud to share some exciting news: our very own President and CEO, Pat Furlong, has been named the 2024 recipient of the prestigious Sonia Skarlatos Public Service Award by the American Society of…

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Updates from the ACHDNC Meeting and Next Steps for Duchenne Newborn Screening

Members of the PPMD community provided testimony today during the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) quarterly meeting, voicing support for adding Duchenne to the Recommended Uniform Screening Panel (RUSP) for…

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