Parent Project Muscular Dystrophy

Wave Life Sciences recently joined PPMD for a community webinar to provide an update on the company’s Phase 2 FORWARD-53 trial of WVE-N531, Wave’s #exonskipping oligonucleotide being investigated for the treatment of individuals with #Duchenne who are amenable to exon 53 skipping. Watch the recording here: https://lnkd.in/eCUgvjVU

From flexible phone mounts that attach to your wheelchair, to voice-activated door knobs, PPMD's #KnightHacks is here to help make everyday tasks more manageable for people living with #Duchenne and #Becker. Explore these technology and equipment resources to help build independence and confidence: https://lnkd.in/etHTj_C8

Yesterday, we received news that the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) has been terminated, effective immediately. PPMD has spent over a decade advancing newborn screening for Duchenne. While this decision creates uncertainty, our commitment remains firm. We are actively working to find alternative pathways to ensure every baby has access to early diagnosis of #Duchenne. Learn more here: https://lnkd.in/eR5BeBks #Duchenne #NewbornScreening #Advocacy

This past weekend, PPMD was joined by families, individuals with Duchenne and Becker, clinicians, researchers, industry partners and local vendors for 2 days of community building, knowledge sharing, and meaningful connections at our PPMD Together event in Minneapolis, MN. Learn more about this event here: https://lnkd.in/eG86x5sk and mark your calendars for our next PPMD Together event in Charlotte, NC September 19-20, 2025.

Avidity Biosciences, Inc. recently joined PPMD for a community webinar to share topline data from their EXPLORE44® clinical trial of del-zota in people living with #Duchenne who are amenable to exon 44 skipping. Learn more and watch the recording: https://lnkd.in/e52ChGx8

PPMD is deeply disappointed that Dr. Peter Marks has made the decision to leave his role as Director of the FDA’s Center for Biologics Evaluation and Research (CBER). Dr. Marks has been an extraordinary public servant and a steadfast advocate, and we are deeply grateful for his years of service. PPMD remains steadfast in our commitment to protecting and advancing the progress we’ve fought so hard to achieve, and we will work closely with regulatory experts, policymakers, and partners to understand the full implications of this personnel change. Learn more: https://lnkd.in/eBpgzSeU

Tune in this afternoon at 2:00 PM ET for a webinar with Avidity Biosciences, Inc. to dive into topline data from their EXPLORE44 trial. There's still time to register and submit questions in advance!

Please join PPMD and Wave Life Sciences for a community webinar on Tuesday, April 1, 2025 at 1:00PM ET. The Wave team will provide an update on the company’s Phase 2 FORWARD-53 trial of WVE-N531, Wave’s exon skipping oligonucleotide being investigated for the treatment of individuals with #Duchenne who are amenable to exon 53 skipping. Wave will discuss the recently released FORWARD-53 data and answer questions from the community. Register today and submit your questions in advance: https://lnkd.in/ePWjCdb8

Wave Life Sciences has announced encouraging interim results from its Phase 2 FORWARD-53 clinical trial of WVE-N531, an exon skipping oligonucleotide being investigated for the treatment of individuals with #Duchenne who are amenable to exon 53 skipping. According to Wave, FORWARD-53 achieved all trial goals, demonstrating sustained exon skipping, muscle concentrations, and dystrophin restoration through 48 weeks. Learn more: https://lnkd.in/eGX2Dp4s

Join PPMD and Avidity Biosciences, Inc. for a community webinar on Friday, March 28, 2025 at 2:00 PM ET. Avidity will share topline data from their EXPLORE44® clinical trial, which is assessing the safety and efficacy of the investigational therapy del-zota in people living with #Duchenne who are amenable to exon 44 skipping. Register and submit your questions in advance: https://lnkd.in/eXju9auT