Parent Project Muscular Dystrophy

Happy Thanksgiving from PPMD! "This Thanksgiving, my heart fills with gratitude for the families who have been with us since the start and those who are just finding their way to PPMD, the dedicated and compassionate healthcare providers, our industry partners and researchers developing the treatments of the future, and our generous donors who support our mission." – Pat Furlong, Founding President & CEO Read more from Pat: https://lnkd.in/eEEhEpiD

Living with #Duchenne and #Becker presents unique challenges, especially for families from underserved or marginalized communities. Health disparities, delayed diagnoses, and limited access to care and clinical research create barriers that often result in diminished clinical outcomes. By working together across various sectors, we can make meaningful strides in improving healthcare & clinical trial access and support for families, no matter their background. Learn more: https://lnkd.in/euetqG5y

ChildMuscleWeakness.org is a comprehensive resource designed to support healthcare providers in identifying pediatric muscle weakness and signs of neuromuscular disease. The Child Muscle Weakness site provides education about recognizing the signs of motor delay and providing the appropriate evaluation and testing. Additional resources include a helpful Motor Delay Algorithm, an extensive video library, and materials for families. Learn more: https://lnkd.in/eX_JqtBb

Recently REGENXBIO joined PPMD for a community webinar to provide an update on its AFFINITY DUCHENNE trial of RGX-202, the company’s microdystrophin #genetherapy for #Duchenne. Watch the webinar recording now: https://lnkd.in/dr27rebn

This 30th anniversary milestone year for PPMD has been marked by significant accomplishments and impactful initiatives. PPMD commemorated the end of the year with “Investing in Every Future”, an event of thanks and celebration in New York City! This meaningful gathering honored three decades of progress and the resilience and spirit of the Duchenne and Becker community, and demonstrated the our commitment to fighting for every future. Looking ahead, we remain steadfast in our mission to accelerate research, shape policy, advance care, empower families, and connect the #Duchenne and #Becker community. Thank you for fighting for every future with us! https://lnkd.in/dRMMJNxr

REGENXBIO Inc. has?announced?that the AFFINITY DUCHENNE? open-label trial of RGX-202 has advanced to pivotal stage and dosed its first patient. The company also announced new, positive efficacy and safety data from the Phase I/II portion of the study, including the first functional data.?PPMD is pleased to learn this update from REGENXBIO and we look forward to learning more about the ongoing AFFINITY DUCHENNE trial during our?upcoming community webinar?on?Wednesday, November 20th, 2024, at 1:00 PM ET. Learn more and register for the webinar here: https://lnkd.in/ec2Krc35

Solid Biosciences joined PPMD earlier this week for a community webinar to provide an update on the INSPIRE DUCHENNE clinical trial for SGT-003, a next-generation microdystrophin gene therapy for individuals living with Duchenne. Included in the update were details about the current progress in the study, the company’s efforts underway to expand eligibility criteria and participating clinical trial sites, and a Q&A portion with the community. Watch the recording: https://lnkd.in/eDs3EdvX

Please join PPMD and REGENXBIO on Wednesday, November 20th at 1 PM ET for a community webinar, “RGX-202: AFFINITY DUCHENNE Pivotal Program and Functional Data.” REGENXBIO will provide an update on its AFFINITY DUCHENNE trial and share interim clinical data for RGX-202, a next-generation microdystrophin gene therapy. PPMD's VP of Clinical Care & Education, Rachel Schrader, MS, APRN, CPNP-PC, and REGENXBIO’s Dr. Naz Dastgir will discuss the company’s pivotal trial plans, explore the first functional data for RGX-202, and offer the Duchenne community an opportunity to ask questions. Register today and submit your questions in advance: https://lnkd.in/effJ_vGH

We’re looking forward to our virtual PPMD Together event, taking place Monday, November 18, 2024 at 7-9 PM ET, as we continue reflecting on 30 years of progress. Join us as together, we share experiences and shape a future where every individual affected by #Duchenne and #Becker can thrive. Connect with the community virtually on November 18, 2024 for an evening of gratitude, connection, and progress—from wherever you are! Learn more and register now: https://lnkd.in/eANK4jHv

Yesterday, REGENXBIO?shared?important updates on their Phase I/II AFFINITY DUCHENNE? trial of RGX-202 for #Duchenne during their third quarter financial results update. We are looking forward to our community webinar with REGENXBIO on Wednesday, November 20 at 1 PM ET to discuss these updates further. Learn more and register today: https://lnkd.in/eNEEwx9q