Parent Project Muscular Dystrophy

Join PPMD and Solid Biosciences on Tuesday, November 12 at 1 PM ET for a community webinar, “INSPIRE DUCHENNE Clinical Trial Update and Study Expansion.” Solid will 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 will be details about the current progress in the study and efforts underway to expand eligibility criteria and participating clinical trial sites. Register today and submit questions in advance: https://lnkd.in/eVc8kRVz

Despite the availability of clinical lab tests and genetic testing, there is an average 2.5-year delay between onset of symptoms and time to diagnosis for patients with #Duchenne. PPMD has partnered with The France Foundation on a comprehensive digital resource library designed for #pediatric and #primarycare practices, featuring micro-learning modules designed to improve awareness and knowledge of the consequences of delayed diagnosis and care initiation in Duchenne patients. Learn more: https://lnkd.in/ei9DDwaX

Tune in TOMORROW at 1:00 PM ET to hear from Satellos Bioscience about the discovery behind SAT-3247. Register now: https://lnkd.in/e9DBu4BN

Join us at 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. Together, we will share experiences and shape a future where every individual affected by #Duchenne and #Becker can thrive. We look forward to connecting with you 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

Join PPMD and Satellos Bioscience for a community webinar on Wednesday 10/30 at 1 PM ET, as we explore a new approach for the treatment of #Duchenne. Satellos will present the discovery behind SAT-3247, a small molecule drug that mobilizes the body’s own muscle stem cells to repair and regenerate muscles. Learn more about the scientific background, mechanism of action, and preclinical data showing promise for this potential new therapy for Duchenne. Register now and submit questions in advance. https://lnkd.in/eY92_JNw

Roche recently joined PPMD for a webinar to share learnings around the natural history of bone health in #Duchenne and detail how the SHIELD study will explore the potential of satralizumab to target both bone health and muscle function in Duchenne. Watch the recording: https://lnkd.in/eQxTb6Nh

PPMD is here for you. In times of emergency, PPMD remains dedicated to providing reliable and timely information to the community. While each family’s situation is unique, here are some helpful resources to reference if your family is navigating an emergency, whether due to illness, injury, or natural disaster. If you aren’t sure where to find answers to your questions, the PPMD Care Team may be able to help. Email us at [email protected] or visit parentprojectmd.org/foryou. CDCC Emergency phone list: https://lnkd.in/eSSitnwF   THINK Resources (for medical emergencies): https://lnkd.in/eAWN3hej   CDC Emergency Preparedness for People with Disabilities: https://lnkd.in/gnp9KzTK

Milestone Moments: PPMD Launches Electronic Health Record Study (2022) In 2022, PPMD launched a study to combine data from The Duchenne Registry with Electronic Health Record (EHR) data from clinics in one central place, the Duchenne Outcomes Research Interchange. By sharing this information, our goal is to help clinicians improve and refine the standards of care, and help researchers learn more about #Duchenne so treatments can be developed faster. Learn more about PPMD’s EHR Study: https://lnkd.in/eUeK_2RZ ___________________________ 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/ee5AwHH2

Calling all citizen scientists! As part of PPMD’s ongoing efforts to better understand and optimize #Duchenne and #Becker care, we are partnering with gastrointestinal (GI), urinary (GU), and swallowing experts to conduct a survey through The Duchenne Registry on patient experiences and preferences. If you aren’t already part of the Registry, join today and help fuel the fight to end Duchenne. Registry participants, please keep an eye out for an emailed invitation to take this important survey. We want to hear from YOU as we navigate the evolution of GI, GU, and swallowing care for individuals living with Duchenne and Becker. https://lnkd.in/g2-8UUTG

Wave Life Sciences recently joined PPMD for a presentation of interim results from the company’s ongoing Phase 2 FORWARD-53 study of WVE-N531 in individuals living with #Duchenne muscular dystrophy who are amenable to exon 53 skipping. Watch the recording: https://lnkd.in/dxx_grkW