Center for Duchenne Muscular Dystrophy at UCLA

13/04/2026

Don't forget to sign up for MDA/Painted Turtle Camp summer session. Applications are due June 12 and camper spots fill fast!
https://www.thepaintedturtle.org/campers-families/summer-session-muscular-dystrophy-leadership?fbclid=IwY2xjawRJTRFleHRuA2FlbQIxMQBzcnRjBmFwcF9pZBAyMjIwMzkxNzg4MjAwODkyAAEeY3EXldMBBM2wJWclSi-aud0eiXw7wrgK7WB2UkWLgFP4F8S2kTSyhe-IVvA_aem_1ktkCEWBqXxSCRb7dl1nhA

Camper Application Deadline: June 12, 2026 | Volunteer Arrival: July 11, 2026

06/04/2026

https://www.cbsnews.com/news/kids-always-getting-sick-lad-1-gene-therapy-ucla-clinical-trial/

Alicia and Jon Langenhop's three children were each diagnosed with a rare disorder. A clinical trial was "a no-brainer."

03/04/2026

Parent Project Muscular Dystrophy will host a webinar on Wednesday, April 8 at 10:30 AM PT to help the community better understand the regulatory context of recent developments with PTC Therapeutics’ withdrawal of the ataluren New Drug Application (NDA) and what this may mean moving forward.
Register here:
https://www.parentprojectmd.org/events/webinar-ataluren-and-the-current-regulatory-landscape/?fbclid=IwY2xjawQ9HpFleHRuA2FlbQIxMQBzcnRjBmFwcF9pZBAyMjIwMzkxNzg4MjAwODkyAAEe3h3UsTU9-CGL14ubwwlSfKeL7tLvRVsILFEDRi0w7cKppMsZZtz-3BZUMo4_aem_G88HR_zyiebuk8QjT4qgkg

Please join PPMD for a webinar on Wednesday, April 8 at 1:30 PM ET to help the community better understand the regulatory context of recent developments with PTC Therapeutics’ withdrawal of the ataluren New Drug...

03/04/2026

PTC Therapeutics has issued the following update on its drug, ataluren, for the treatment of DMD with nonsense mutations. Little Hercules has reached out to PTC with questions. Drop any of yours in the comments and we will do our best to get answers.

01/04/2026

"Clinical trial participants are heroes whose bravery helps the entire disease community have access to improved therapies.”

First therapy funded by California taxpayer dollars is approved to treat rare childhood disorder FOR IMMEDIATE RELEASE Contact: Amy Adams Senior Director of Communications press@cirm.ca.gov South San Francisco, Calif., March 27, 2026 – The California Institute for Regenerative Medicine (CIRM) cele...

30/03/2026

Join Parent Project Muscular Dystrophy on April 1st 10am PT to learn more about the expanded Duchenne Natural History Study (eDNHS)—a global observational study designed to deepen our understanding of Duchenne muscular dystrophy in today’s therapeutic environment.
This webinar will give the community the opportunity to learn about the purpose of the study, how it is designed, how you can participate and why this study is so important for the future of Duchenne therapy development and care.

Register here:
https://www.parentprojectmd.org/events/webinar-understanding-the-expanded-duchenne-natural-history-study-ednhs-2/?fbclid=IwY2xjawQ3sxxleHRuA2FlbQIxMABicmlkETFvazRuQzdPTkhma3pnUnhyc3J0YwZhcHBfaWQQMjIyMDM5MTc4ODIwMDg5MgABHnCZAenZXrMTGyOkhQ9tBvRsEanoiQDi3BAGHO_KtV0PqPzeVqIkXof31A4g_aem_ScyWti9kKheCjBKBqfNikg

Join us to learn more about the expanded Duchenne Natural History Study (eDNHS)—a global observational study designed to deepen our understanding of Duchenne muscular dystrophy in today’s therapeutic environment. Building on the original Duchenne Natural...

20/03/2026

From Action Duchenne
Register here: https://www.actionduchenne.org/in-person-support-events/?fbclid=IwY2xjawQqgoZleHRuA2FlbQIxMABicmlkETFqb3I3cUFmbGFxRmlYU24xc3J0YwZhcHBfaWQQMjIyMDM5MTc4ODIwMDg5MgABHl6MpE79eCOmB6ulXrky-fiJui_hdEAVJEscuLQB68LE69UkTJjdHc4yLDL1_aem_4wtqD-3t1nocEyXMMPUlDg

20/03/2026

Scholarships available from the EveryLife Foundation for Rare Diseases!
The Scholarship Fund is now accepting applications. This year, 58 scholarships of $5,000 will be awarded to individuals living with a rare disease. The scholarship is open to individuals ages 17 and older in the United States who are enrolling in college, graduate school, or a trade program for Fall 2026.
More details and Apply here:

19/03/2026

Muscular Dystrophy Association Quest magazine product guide recommended by MDA ambassadors who use them.

19/03/2026

CureDuchenne futures is a great conference with lots of fun things for adults, kids and siblings.

FUTURES 2026 | May 20–24 | Orlando, Florida

It's almost time! 💙

We’re counting down the days until we come together in Orlando, Florida | May 20–24 for a powerful week of connection, learning, and progress for the Duchenne community. From groundbreaking science to meaningful moments with families, advocates, and researchers—FUTURES is where our community moves the future forward.

We can’t wait to see you there. Who’s joining us? ✨

Learn more and register using the link below:
https://cureduchenne.org/event/2026-futures-national-conference/

19/03/2026

03/19/26
Sarepta Therapeutics Provides Regulatory Update on AMONDYS 45® and VYONDYS 53®
- Following feedback from FDA, Company intends to submit supplemental new drug applications to FDA by the end of April 2026 requesting conversion to traditional approval.
Full PR here: https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-provides-regulatory-update-amondys-45r-and-vyondys-53r

19/03/2026

Sarepta Therapeutics Announces that Screening and Enrollment are Underway in ENDEAVOR Cohort 8 to Evaluate Enhanced Immunosuppression Regimen as Part of ELEVIDYS Gene Therapy for Non-Ambulant Individuals with Duchenne.
Full PR Here: https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-announces-screening-and-enrollment-are-underway-endeavor