Center for Duchenne Muscular Dystrophy at UCLA

01/10/2026

Applications are now open for MDA's Summer 2026 Season.
Info here:

As Muscular Dystrophy Association looks ahead to its next 75 years, the 71st MDA Summer Camp session continues a legacy of empowering the next generation of youth living with neuromuscular conditions.

01/07/2026

Thinking about everyone affected by the fires last year. We love our city and community.

One year later, UCLA Health experts say long-term threats to lung health, mental well-being and overall community wellness remain.

12/22/2025

Wishing everyone a very Happy Holiday Season. And sending out a huge Thank YOU for all the end of year donations thus far. We cannot express how much we appreciate your continued support. We absolutely cannot continue our work without support from private donations, this year has been especially challenging for research funding and we are more than grateful for any amount of support you can give. It's not too late if you still have room in your budget!

12/17/2025

Thank you again to all of the organizations and advocates who helped get this done! A huge win for the DMD and MLD patient and scientific communities.

🎉 Exciting Newborn Screening Update! Today we are celebrating a life-saving advancement!

The U.S. U.S. Department of Health and Human Services (HHS) has added Duchenne muscular dystrophy (Duchenne) and Metachromatic Leukodystrophy (MLD) to the federal Recommended Uniform Screening Panel (RUSP).

This is a significant milestone for public health, opening the door to more timely interventions and optimized health outcomes for children affected by these rare diseases.

We congratulate the rare disease advocates and public health officials who worked tirelessly to make today’s progress possible—and we especially recognize and honor the Duchenne and MLD communities and the patient advocacy organizations who never gave up, especially the condition nominators: Parent Project Muscular Dystrophy, MLD Foundation, and Muscular Dystrophy Association.

Thank you to Secretary Kennedy and HHS for demonstrating a strong commitment to newborn screening and the federal RUSP.

To read the full story and learn more visit: https://everylifefoundation.org/hhs-adds-duchenne-muscular-dystrophy-and-mld-to-the-federal-recommended-uniform-screening-panel-rusp/

Pictured below: Annie Kennedy, senator Roger Wicker, Pat Furlong, Congressman Gus Bilirakis

12/16/2025

Capricor and PPMD Webinar is tomorrow.

– Webinar to be held Wednesday, December 17, 2025, at 1:00 p.m. ET – SAN DIEGO, Dec. 16, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics,…...

12/16/2025

Today marks a monumental victory for the Duchenne community. The U.S. Department of Health and Human Services (HHS) has officially added Duchenne to the Recommended Uniform Screening Panel (RUSP)—the national list of conditions recommended for...

12/16/2025

Really great news!! Thanks to all the amazing people and organizations that made this possible.

Parent Project Muscular Dystrophy and Muscular Dystrophy Association Celebrate Monumental Victory for Duchenne Muscular Dystrophy

12/16/2025

PPMD will host a community webinar with Capricor Therapeutics on Wednesday, December 17 at 1:00 PM ET as they share their most recent results from the Phase 3 HOPE-3 trial evaluating deramiocel (CAP-1002), the company’s investigational cell therapy for the treatment of Duchenne muscular dystrophy. The presentation will include a review of the data, a discussion of planned next steps for FDA engagement and regulatory strategy, and a dedicated Q&A period.
Register and submit questions here:
https://www.parentprojectmd.org/events/webinar-capricor-therapeutics-hope-3-community-update/?fbclid=IwY2xjawOt19NleHRuA2FlbQIxMQBzcnRjBmFwcF9pZBAyMjIwMzkxNzg4MjAwODkyAAEeRUxmulJkktzWk8wQ4OoFE6U-_Drz2fuyDiqKHvrGGSmiMWHhHdPk09SIwhY_aem_dC77sdu-4Ah3ScekbXo3sw

Please join us for a community webinar with Capricor Therapeutics on Wednesday, December 17 at 1:00 PM ET as they share their most recent results from the Phase 3 HOPE-3 trial evaluating deramiocel (CAP-1002), the...