Parent Project Muscular Dystrophy

Milestone Moments: PPMD Expands Cardiac Initiative with $2 Million Grant (2022) PPMD has an ongoing commitment to cardiac care, because the heart is a muscle too. As an organization, we have heavily invested in the pursuit of optimal care, interventions, and research pertaining to the management and prevention of cardiomyopathy. In 2022, we announced our commitment of $2 million to support the Advanced Cardiac Therapies Improving Outcomes Network (ACTION). Overall, PPMD has invested nearly $7 million to manage and prevent heart failure in #Duchenne through our Cardiac Initiative. We have learned so much, but there is still a tremendous amount to do to understand and improve heart health in people with Duchenne. For this reason, PPMD is committed to continuing to bring together experts to understand cardiac needs and opportunities; further exploring novel cardiac treatments; and understanding more about device and transplant interventions. PPMD recently held the 2024 Cardiac Care Workshop, convening academics, healthcare professionals, and industry partners to discuss how to move beyond the current state of cardiac care to accelerate research, advance standards of care, and better understand Duchenne and Becker in a rapidly evolving and complicated landscape. During this third meeting in PPMD’s three-part Cardiac series, attendees took a deeper dive into the rapidly evolving landscape of approved therapy and post-gene therapy cardiac monitoring and lifespan cardiac care. Learn more: https://lnkd.in/eHdtjcVs -------- As we reflect on the progress we’ve made as a community since PPMD’s founding in 1994, we’re sharing milestone moments in the fight for every future from the last three decades. #PPMDEveryFuture Learn how you can join PPMD in the Fight for Every Future: https://lnkd.in/ejAWUnip

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 making its way to President Biden's desk to be signed into law, represents a groundbreaking advancement in air travel accessibility, particularly for individuals living with disabilities. Learn more about this important legislation and its implications for the #Duchenne and #Becker community: https://lnkd.in/eKVbwHR6

Yesterday, PPMD’s President & CEO, Pat Furlong, spoke during a Women's International Forum event held at the United Nations on “Ethics, Advocacy, and Global Impact: A Multidisciplinary Dialogue with Leaders in Medical Ethics, Muscular Dystrophy Research, and International Diplomacy”. During her presentation, Pat highlighted the importance of #Duchenne #newbornscreening, as well as access to diagnosis, care, therapies, and clinical trials.

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 care to accelerate research, advance standards of care, and better understand #Duchenne and #Becker in a rapidly evolving and complicated landscape. We are eager to see the impact of PPMD’s Cardiac Care Workshop series over the last three years. With a clear vision moving forward we hope to create a path ahead for aggressive, proactive cardiac care for all individuals living with dystrophinopathy. To hear more about the Cardiac Care Workshop, join us at PPMD’s 30th Annual Conference, taking place June 27-29, 2024 in Orlando, Florida. We hope to see you there – register today to join us in person or virtually!! We look forward to sharing more detail about this meeting with the community, including an opportunity to engage and ask questions. https://lnkd.in/eHdtjcVs

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 Gene & Cell Therapy. This esteemed award recognizes individuals who have demonstrated outstanding dedication to public service in the field of gene and cell therapy. Pat’s unwavering commitment to advancing research, impacting policy, improving care, and supporting families affected by #Duchenne and #Becker embodies the spirit of this award. Congratulations, Pat, on this well-deserved honor! https://lnkd.in/gabYEVQT

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 #newbornscreening. In advance of the meeting, PPMD and the Muscular Dystrophy Association, as the nominators of the Duchenne package, requested that the committee delay the vote on adding Duchenne to the RUSP until later in the year, as crucial data that may support the package is anticipated to become available for the committee’s consideration in the near future. Today, the ACHDNC Committee unanimously voted to postpone its decision for up to one year on whether to recommend Duchenne for inclusion on the RUSP until additional evidence is available to make a decision. PPMD is actively working with key opinion leaders and clinicians to bring forth additional data and we anticipate learning more from the states who have and will begin screening. We are appreciative to the ACHDNC for recognizing our request for a delay to allow for a more comprehensive consideration of additional data, and extend our heartfelt gratitude once again to the families and experts who shared their testimonies today. Learn more: https://lnkd.in/es7gtZzC

PPMD is excited to bring together experts in the cardiac field, spanning clinicians and researchers, to the third annual PPMD Cardiac Workshop, May 9-10, 2024 to delve into current and emerging issues in cardiac care in #Duchenne and #Becker. Learn more and stay tuned — following the Workshop, PPMD will share updates with the wider Duchenne community in a comprehensive recap blog highlighting key learnings from the meeting. https://lnkd.in/eMXn-xV8

PPMD advocates Marit and son Brecken met with members of the Minnesota House & Senate legislature yesterday at the State Capitol in Saint Paul, MN. We’re thrilled to share that proclamation language recognizing September 7th as #Duchenne Awareness Day in Minnesota has officially been introduced in the Minnesota House of Representatives!

We are devastated to learn of the tragic loss of a young boy that was participating in Pfizer’s Phase 2 DAYLIGHT study. This study is evaluating the safety and dystrophin expression of fordadistrogene movaparvovec (PF-06939926), Pfizer’s gene therapy candidate for #Duchenne, in boys who are two and three years old. The company issued a community letter sharing this devastating news and that it is committed to sharing more information as it becomes available. We anticipate additional updates from Pfizer as the company’s investigation continues. Our hearts ache for this family, and this loss underscores the critical importance of understanding what transpired to ensure the safety of all individuals participating in clinical trials. As a community, it is imperative that we come together to support each other during this time and work collectively towards making advancements in research while prioritizing safety above all else. Read more, including a note from PPMD’s Pat Furlong: https://lnkd.in/eFPg2dkM