September 17, 2024

Navigating Access & Resources

Navigating Access & Resources Navigating insurance access for medical care such as approved therapies or equipment can be daunting. Parents, patients, and healthcare providers spend hours putting together documents and speaking on the phone trying…

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September 12, 2024

Watch the Recording: Advocating for A Successful School Year

Recently, PPMD’s Alexis Hazlett was joined by panelists Jodi Krause (Colorado Children’s Hospital), Jill Castle, and Patrick Moeschen to discuss advocating for your child throughout the school year. The speakers dove into the differences between…

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September 9, 2024

Setting Up A Successful School Year

Setting Up A Successful School Year PPMD is here to make sure that families within the Duchenne and Becker community have the necessary tools to advocate for their child not only in medical settings, but…

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September 6, 2024

Raising Our Voices for Duchenne: Recap of PPMD’s Advocacy Webinar

On Thursday, September 5, 2024, PPMD hosted a special webinar, “PPMD Advocacy: Raising Our Voices in 2024 and Beyond,” bringing together advocates to reflect on PPMD’s advocacy efforts in our 30th anniversary year and discuss…

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September 5, 2024

Myology Course Educates and Advances Next Generation of Neuromuscular Physicians and Scientists

PPMD is dedicated to fighting for every future as we strive to end Duchenne. Since 2011, PPMD has been a proud supporter of the Myology Course, presented by Nationwide Children’s Hospital (NCH) and The Ohio…

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August 20, 2024

WATCH: Advancing DYNE-251 – An Investigational Medicine for Duchenne to Deliver for Patients (Webinar Recording)

Dyne Therapeutics recently joined PPMD for a presentation of clinical data from the DELIVER Trial of DYNE-251 in individuals living with Duchenne muscular dystrophy who are amenable to exon 51 skipping, first released on May…

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August 13, 2024

Finding Support to Manage Behaviors in Duchenne and Becker

Research has shown that dystrophin is usually present in the brain as well as in the muscles. Scientists believe that when dystrophin is missing or partially functional, like in Duchenne and Becker, brain neurons may…

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August 12, 2024

PPMD Urges FDA to Conduct a Full Review of Ataluren (Translarna) Following Resubmission of NDA

PTC Therapeutics has shared that it has resubmitted its New Drug Application (NDA) to the FDA for review and potential approval of ataluren (Translarna). Ataluren is an oral small molecule treatment that allows for stop-codon…

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August 9, 2024

WATCH: Edgewise Therapeutics – Clinical Trials in Becker Muscular Dystrophy (Webinar)

Edgewise Therapeutics recently joined PPMD for a webinar to provide the Becker community information about the company’s clinical trials for Becker. Edgewise’s Chief Scientific Officer and Chief Medical Officer, shared two year results from the…

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August 9, 2024

Avidity Biosciences Announces Positive Data From Phase 1/2 EXPLORE44™ Trial of AOC 1044 in Individuals Amenable to Exon 44 Skipping

Avidity Biosciences, Inc. today announced positive data from the company’s Phase 1/2 EXPLORE44™ clinical trial of AOC 1044, an exon skipping therapy targeting those amenable to exon 44 skipping. Avidity also announced the approved international…

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