Center for Duchenne Muscular Dystrophy at UCLA

09/07/2025

https://www.parentprojectmd.org/make-your-voice-heard-support-adding-duchenne-to-the-rusp/?fbclid=IwY2xjawMqDEFleHRuA2FlbQIxMQABHjfqtrOntaIj8_b8Ns0ATM4iT5SCSgW4ns3BoUE2PohLDEQG8QW-c97zY4OR_aem_qkCPNXZbZ2aR96WnjvYEEQ

Every year, thousands of newborns in the U.S. are screened for serious conditions whose early detection can change the course of their lives. Right now, we have a chance to ensure Duchenne muscular dystrophy is...

09/07/2025

Joining with MDA to support World Duchenne Awareness Day.

09/07/2025

Joining CureDuchenne to celebrate Duchenne Awareness Day!

09/07/2025

Joining PPMD in celebrating Duchenne Awareness Day!

09/07/2025

Today is Duchenne Awareness Day. Signs of DMD usually are recognized when kids are very young. However, diagnosis often takes place at the age of 4 or even later. Symptoms include: muscles getting weaker over time, trouble walking, falling down a lot, and being slower to reach functional milestones like crawling or walking.
Watch World Duchenne Organization's video 'What is Duchenne" here:
https://vimeo.com/133337162

09/05/2025

A big Congratulations to the 25 recipients of Route 79, The Duchenne Scholarship Program from Sarepta!
"Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced 25 recipients of Route 79, The Duchenne Scholarship Program, for the 2025-2026 academic year. Of the academic scholarships, 20 will be awarded to individuals living with Duchenne and five to siblings of individuals living with Duchenne."
Full PR with recipients names here: https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-recipients-route-79-duchenne-4

09/05/2025

Check out Dr. Nelson and Dr Miceli, our CDMD Co Directors and 'Cellular Pioneers,' featured in Rare Revolution Magazine.
https://urldefense.com/v3/__https://bit.ly/Cellular-Pioneers__;!!F9wkZZsI-LA!GZhRkypB4tXVZLceNzAo2CYouy7qvKVSgoPN0owuAv-Xtg7Y3AGXoRD6sxrBraANm4E1jjvnWxkGjJv2-kL5ulMRCB1KzxM$

09/03/2025

September is Duchenne Awareness Day, Duchenne Action month, and Newborn Screening Awareness month. In April of 2025, the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) was disbanded. This was the route previously used to get conditions added to the Recommended Uniform Screening Panel (RUSP). Duchenne had already completed their evidence review process and was scheduled for consideration at the time of the ACHDNC’s elimination. Thus, the path to the federal Recommended Uniform Screening Panel was uniquely disrupted.

To address this unique circumstance, HHS has been working with the Health Resources Services Administration (HRSA) to ensure a path forward. HRSA has supported the completion of the evidence review and will be making it publicly available. To complement the completion of the evidence review, the EveryLife Foundation is providing a forum for the evidence to be presented to the community and discussed by newborn screening experts. The EveryLife Foundation for Rare Diseases is hosting a virtual Newborn Screening Evidence Review Workshop on September 12, 8am PT.
Register here: https://us02web.zoom.us/webinar/register/WN__UWTxsCjTHOhPlkexvps7g #/registration

https://everylifefoundation.org/newborn-screening-take-action/newborn-screening-evidence-review-workshop/

09/03/2025

World Duchenne Awareness Day is Sunday, September 7. September is also Newborn Screening awareness month. The U.S. Department of Health and Human Services (HHS) is currently reviewing the nomination to add Duchenne to the RUSP and has requested public comments. We support Parent Project Muscular Dystrophy years long effort to add Duchenne to the Recommended Uniform Screening Panel so that all babies born with Duchenne have prompt access to information and available therapies and no longer wait years for a diagnosis. Please share your story or submit comments here:

Every year, thousands of newborns in the U.S. are screened for serious conditions whose early detection can change the course of their lives. Right now, we have a chance to ensure Duchenne muscular dystrophy is...