Center for Duchenne Muscular Dystrophy at UCLA

07/11/2026

Recordings and resources from PPMD's 2026 Annual Conference are now available! Thank you Parent Project Muscular Dystrophy.

Whether you joined us in Orlando or are catching up from home, the learning opportunities from PPMD’s 2026 Annual Conference continue. We’re excited to share that recordings and resources from PPMD’s 2026 Annual Conference are...

07/08/2026

From ITF Therapeutics Presenting new data on Givinostat in Duchenne.
"Quantitative MRI measures of DMD disease progression from the Phase 3 EPIDYS trial were associated with statistically significant differences between treatment group vs. placebo in measures of muscle composition, including contractile cross-sectional area (cCSA) and fat fraction, across selected lower limb muscles1"
Full PR here:

Phase 3 EPIDYS MRI data quantify givinostat’s potential to preserve functional muscle tissue and reduce fat infiltration in patients with Duchenne muscular...

06/30/2026

Sarepta today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the supplemental New Drug Applications (sNDAs) for AMONDYS 45® (casimersen) and VYONDYS 53® (golodirsen) for the treatment of Duchenne muscular dystrophy (DMD). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action data of February 28, 2027.

These submissions seek conversion of the accelerated approvals of AMONDYS 45 and VYONDYS 53 to traditional approvals.

The applications are supported by data from the ESSENCE confirmatory study, as well as substantial published real-world evidence and the favorable and consistent safety profiles of both exon-skipping therapies.
More info here:
https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-announces-fda-acceptance-sndas-amondys-45r-and-vyondys

06/30/2026

Attention Duchenne advocates! Please join this Important community webinar with Parent Project Muscular Dystrophy to learn and prepare for the upcoming FDA Advisory committee meeting on Capricor Therapeutics, Inc. Deramiocel.

Parent Project Muscular Dystrophy (PPMD), in collaboration with Duchenne advocacy partners Best Day Ever Foundation, CureDuchenne, Jett Foundation, Kindness Over Muscular Dystrophy, Inc., Little Hercules Foundation, Muscular Dystrophy Association, Muscular Dystrophy Family Foundation, and Team...

06/29/2026

“Fast Track designation represents an important validation of SAT-3247 and our commitment to transforming the treatment landscape for Duchenne,” said Frank Gleeson, co-founder and chief executive officer of Satellos. “Together with our Orphan Drug and Rare Pediatric Disease designations, this recognition further strengthens the momentum behind our clinical program. We believe SAT-3247’s unique regenerative mechanism has the potential to address a fundamental aspect of disease progression by re-establishing the biological signals needed for effective muscle repair and regeneration. As we advance our Phase 2 studies, we look forward to continuing our engagement with the FDA as we work to advance SAT-3247 for individuals and families affected by Duchenne.”

Full PR here:

SAT-3247 has now received FDA Fast Track, Orphan Drug and Rare Pediatric Disease designations for Duchenne muscular dystrophy (“Duchenne” or “DMD”) Fast Track is designed to expedite the development/review of new drugs to treat serious or life-threatening conditions and fill unmet medical ne...

06/28/2026

Good read from Pat Moeschen, Member of Parent Project Muscular Dystrophy Adult Advisory Committee.

Being part of the PPMD conference's Adult Advisory Committee has helped columnist Patrick Moeschen bond with those who share his diagnosis.

06/26/2026

Capricor Therapeutics, Inc. FDA Advisory Committee meeting is scheduled for July 29, 2026
Company's Biologics License Application on track with PDUFA target action date of August 22, 2026

–Advisory Committee meeting scheduled for July 29, 2026––Company's Biologics License Application on track with PDUFA target action date…...

06/22/2026

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...

06/18/2026

You can now watch CureDuchenne Futures 2026 sessions on You Tube.

Thank you to everyone who made FUTURES 2026 such a meaningful and inspiring event. 💙

From research breakthroughs and clinical care updates to powerful community stories, the conversations continue beyond the conference.

Missed a session or want to revisit a presentation? Many of our main sessions are now available on YouTube.

🎥 Watch now using the link below:
https://youtube.com/playlist?list=PLGh1VHVmJWeOUNwK4KH0SPMrytOMultr_&si=eGcS-ePkpr5jz3eF

06/10/2026

Parent Project Muscular Dystrophy conference App is LIVE!

📱 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