Parent Project Muscular Dystrophy

ACTION ALERT! Now is the time to make your voice heard. Urge your House and Senate Members to support the FY 2026 #Duchenne funding request, protect Medicaid, and reauthorize the Priority Review Voucher (PRV) program. Please take a moment to send a message to your Members of Congress and emphasize the importance of prioritizing Duchenne and #Becker patient care, public health, and research. Take action now: https://lnkd.in/eWXWzfhY

PPMD, with a group of more than 150 advocates, heads to Capitol Hill today to meet with Congressional leaders during this year’s annual PPMD Advocacy Conference. This marks the 23rd year that PPMD advocates will gather in Washington to urge Congress to advance legislation that will bring us closer to ending #Duchenne. Learn about this year’s Advocacy Conference and our requests to Congress: #EndDuchenne #PPMDAdvocacy

We are on the ground in Washington, DC, for PPMD’s 2025 Advocacy Conference, getting ready to head to Capitol Hill to urge members of Congress to continue making #Duchenne and #Becker patient care, public health, and research a top priority. Today kicks off with a panel discussion about what’s going on in Washington, featuring experts to talk about NIH, DoD, Congress, and federal agencies. Thank you to our speakers: Dave Zook, Nick Manetto, Joel Wood, and John D. Porter. And thank you to our PPMD Advocates, both in DC and at home around the country, for sharing your powerful stories with lawmakers and making success in our community possible. #EndDuchenne #PPMDAdvocacy

Please join PPMD and Capricor Therapeutics, Inc. for a?community webinar?TOMORROW,?Thursday, March 6, 2025 at 1:00PM ET.?The Capricor team will provide a current update on the company’s Biologics License Application (BLA) seeking full approval of deramiocel (CAP-1002), Capricor’s novel cell therapy for the treatment of cardiomyopathy in patients diagnosed with #Duchenne. Register today and submit your questions in advance: https://lnkd.in/ehp3nvFv

PPMD is excited to learn that the?FDA has accepted?for review and granted Priority Review for Capricor Therapeutics, Inc. Biologics License Application (BLA), seeking traditional approval for deramiocel (CAP-1002) for the treatment of #Duchenne cardiomyopathy. Capricor will join PPMD for a community webinar this Thursday, March 6 at 1 PM ET to discuss this update. Learn more: https://lnkd.in/ehCN2rD9

Members of the Solid Biosciences team recently joined PPMD for a community webinar to review initial positive data from the INSPIRE DUCHENNE study of SGT-003, a next-generation microdystrophin gene therapy candidate. Watch the recording: https://lnkd.in/efKtQ-dZ

Today is #RareDiseaseDay, a day to give voice to 300 million people living with rare diseases and their families, through education and advocacy. This year, PPMD participated in #RareDC2025 on Capitol Hill, hosted by the Rare Disease Legislative Advocates (a program of the EveryLife Foundation for Rare Diseases). This multi-day event empowers participants to learn key policies affecting the rare disease community and share their stories with Members of Congress. PPMD Advocates from across the country will be heading to Washington, DC in March for PPMD’s annual Advocacy Conference. Learn more and register today to raise your voice in the fight to #EndDuchenne: https://lnkd.in/dTY83Auy

Staying healthy and feeling your best is important, but managing personal care can come with unique challenges. Check out PPMD’s Knight Hacks for tips and tricks from the #Duchenne and #Becker community on health and personal hygiene—empowering you with real-life strategies for everyday care! https://lnkd.in/eSfPrx_j

This Rare Disease Week, we are sending a petition to Congress to urge Members to continue their support of steady and robust federal agency leadership, federal biomedical research funding, and public health agency resources. #RareDC2025 Take action and help us spread the word:

?? Calling all rare disease community members – patients, caregivers, clinicians, researchers, and advocates! Join a petition to Congress in honor of Rare Disease Week 2025 #RareDC2025 ??? On behalf of our nation’s rare disease community, this petition urges Congress to continue their support of steady and robust federal agency leadership, federal biomedical research funding, and public health agency resources. ??? The Community’s letter will be sent to All Members of Congress on Rare Disease Day, Friday, February 28, so please take action and help spread the word: https://lnkd.in/eJgQQtyT