Apply by this Sunday, August 31st to serve on PPMD's Adult Advisory Committee (PAAC) and lend you time, talent, and vision to paving the pathway forward for the #Duchenne and #Becker community. Learn more about the PAAC and apply today:.

RT @MDAorg: BREAKING NEWS: @MDAorg and @ParentProjectMD have published Consensus Guidelines for safe, equitable delivery of #GeneTherapy in….

What do you want to know?.

Join us for Becker Education & Engagement Day 2025! BEED will be held in Sacramento, Orlando, St. Louis, Philadelphia, and Seattle. Registration is free for those living with #Becker and their families. Register now to secure your place! Learn more:

In welcoming Katherine to work alongside President Pat Furlong, our staff, and the community, we are furthering our mission to end Duchenne, support families, and foster collaboration—ensuring that your voices and needs remain central to every effort.

Katherine brings with her over 15 years of experience in Duchenne and rare disease, a deep respect for clinical science, a heartfelt commitment to our mission, and a remarkable ability to connect across the research, care, and advocacy spaces.

Today, we are excited to share a new chapter in PPMD’s journey! Our leadership team is expanding with the addition of Katherine Beaverson, MS, as Chief Executive Officer.

We’re excited to announce Bad Shirt Friday, a global awareness campaign to help shine a light on #Duchenne. On Friday, September 5th, wear your loudest, most outrageous shirts and share a selfie on social media using the hashtag #BadShirtFriday.

By sharing your story, you can help ensure that no family has to wait for a Duchenne diagnosis. Learn more about submitting written comments—and check out our written comment guide for step-by-step instructions—here:

The U.S. HHS is currently reviewing the nomination of #Duchenne to be added to the RUSP, and we need families, advocates, clinicians, and allies to submit written comments to the Federal Register by September 15, urging HHS to move forward with this lifesaving addition.

Recently @Solid_Bio shared an update on the company’s Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003 for the treatment of individuals living with #Duchenne. Learn more:

PPMD is seeking adult (ages 18 and older) members of our Duchenne and Becker community to serve on the PPMD Adult Advisory Committee (PAAC). Learn more and apply by August 31:

Calling all adults with #Duchenne & #Becker:. Have you been looking for a way to engage with the community more? Do you have a personal experience that you would like to share with the larger community or through federal and state advocacy efforts?.

Registration is now OPEN for our second PPMD Together event of 2025 — join us in Charlotte, NC on September 19-20 for a weekend of gratitude, connection, and progress!

. the HOPE-2 and HOPE-3 (Phase 3) clinical trials, future plans to submit HOPE-3 data to the FDA, and addressed questions from the community. Watch the recording:

Earlier this week @Capricor joined PPMD for a community webinar to discuss the current status of Capricor’s BLA for Deramiocel. We discussed the regulatory implications of a CRL, as well as provided information about cardiomyopathy in Duchenne, . .

The FDA has announced that the agency is now recommending the removal of @Sarepta voluntary hold for ambulatory patients eligible to receive ELEVIDYS. Sarepta will resume shipping ELEVIDYS to sites of care imminently. Learn more:

Today @aviditybio shared that the FDA has granted Breakthrough Therapy designation to del-zota (AOC 1044) for the treatment of #Duchenne in individuals amenable to exon 44 skipping. Learn more:

We urge Sarepta and FDA to communicate with urgency, clarity, and transparency, and we're hopeful this pause will provide sufficient time to analyze available data, ensuring patients, families, and clinicians can make informed decisions.

The events of the past several days have been unsettling, and the news that Sarepta has now temporarily halted all shipments, including to the ambulatory population covered under the current traditionally approved label, has understandably intensified concerns and uncertainty.

This evening, @Sarepta issued a press release stating that it has voluntarily and temporarily paused all shipments of ELEVIDYS for #Duchenne in the U.S., effective close of business tomorrow, 7/22/25.