Parent Project Muscular Dystrophy

06/22/2026

Earlier today, PPMD Chief Executive Officer Katherine Beaverson, MS, attended the U.S. Department of Health and Human Services (HHS) Clinical Trials Roundtable in Washington, D.C., joining leaders from across government, industry, academia, and the patient advocacy community.

The roundtable, convened by HHS leadership, focused on a central challenge: how to cut the duration of clinical trials in half while maintaining the highest standards of safety and scientific rigor. The conversation underscored a growing urgency across the federal government to ensure the United States remains the global leader in biomedical research, particularly as more early-stage trials move overseas due to speed and cost.

Learn more about the roundtable and next steps for the Duchenne and Becker community:

On June 22, 2026, PPMD Chief Executive Officer Katherine Beaverson, MS attended the U.S. Department of Health and Human Services (HHS) Clinical Trials Roundtable in Washington, D.C., joining leaders from across government, industry, academia, and...

06/21/2026

❤️ Happy Father’s Day from PPMD! ❤️

We’re celebrating the dads, grandfathers, and father figures in our community who stand beside their families with resilience, dedication, and unwavering love.

Honor the father your life with a gift to PPMD and send him an eCard to show your love and appreciation for all he does: donate.parentprojectmd.org/fathersday

06/18/2026

Today, the FDA released a recap of the Rare Disease Roundtable that PPMD participated in earlier this month.

The discussion focused on advancing patient-centered drug development and strengthening regulatory pathways for rare diseases. PPMD encouraged the FDA to continue utilizing and refining tools already available or currently under development to support the review of therapies for rare disease communities, including and .

We are grateful to the FDA for convening this important conversation and for its continued partnership. We look forward to continued meaningful engagement.

Recently FDA leadership held a roundtable discussion with 15 dedicated rare disease leaders.

06/18/2026

What is it really like to grow up with Duchenne or Becker muscular dystrophy? In this episode of PPMD's Livestream with the PAAC, "A Day in the Life," PAAC members will share personal reflections on childhood, adolescence, and adulthood, including navigating school, gaining independence, building support systems, and adapting to life with a progressive condition. From receiving a first power wheelchair to discovering new opportunities and purpose as adults, panelists offer honest insights, practical advice, and encouragement for the next generation.

Join us for a candid conversation about resilience, community, and what it means to live a full life with Duchenne or Becker. We will also discuss how they are helping shape the future of the muscular dystrophy community and the work they will be leading at PPMD's Annual Conference in Orlando.

This is all about swapping real-life tips and learning from people who’ve been there. Come hang out, ask questions, and pick up a few things you definitely won’t find on Google: LIVESTREAM WITH THE PAAC!

*The livestream will appear directly on PPMD's page when we go live at 1:00 PM ET on June 18, 2026.*

06/11/2026

Season 7 of our Living Duchenne Podcast continues with an important discussion about raising an independent child in the middle years.

Host Rachel Poysky sits down with guests Colleen Labbadia and Kate Vanderweele, who share their experiences parenting in those adolescent and early teen years (middle school and high school ages) with a mindset of preparing their children for a future with independence. We talk through successful practices to build ownership, self-advocacy, and incorporate “low stakes” situations that empower their young boys to autonomy.

Take a listen to Season 7 Episode 2: Raising an Independent Child - The Middle Years, now available on Apple Podcasts, Spotify, or wherever you get your podcasts!

https://www.parentprojectmd.org/care/living-duchenne-podcast/season-7-raising-an-independent-child/

06/09/2026

📱 PPMD's 2026 Annual Conference App is LIVE!

Joining us at Conference later this month? Download the app to customize your Conference experience to dive deeper into the topics you’d like to explore, while connecting with others and recharging with your community.

🗓️ Check out the agenda, start building your own schedule, and book 1-on-1 appointments with experts before we even arrive in Orlando!

If you haven’t registered yet, it’s not too late! Learn more about registering and accessing the app: https://web.cvent.com/event/4c265373-9bde-4c0a-9d7e-922302d52dbd/summary

06/05/2026

📢 As of today, Massachusetts has officially begun screening all newborns for !

We extend our deepest gratitude to the dedicated Massachusetts families, clinicians, and advocates who shared their stories, provided testimony, and championed this amendment. Your unwavering efforts made this victory possible.

Learn more about this milestone, resources available for newly diagnosed families, and how you can get involved in PPMD’s newborn screening advocacy efforts:

As of today, Massachusetts has officially begun screening all newborns for Duchenne muscular dystrophy. This milestone comes after the passage of the state’s Maternal Health bill (H. 4999), which was signed into law by Governor...

06/05/2026

Join PPMD and Edgewise Therapeutics for a community webinar on Wednesday, June 17 at 1:00 PM ET for an update on the sevasemten program in . Edgewise will share the latest updates on the ongoing LYNX and FOX clinical trials, provide context around recent news, and answer questions from the Duchenne community.

Register today and submit your questions in advance:

Join PPMD and Edgewise Therapeutics for a community webinar on Wednesday, June 17 at 1:00 PM ET for an update on the sevasemten program in Duchenne muscular dystrophy. Edgewise will share the latest updates on...