Center for Duchenne Muscular Dystrophy at UCLA

01/10/2025

Medical services will continue. Other help may be delayed, halted

29/09/2025

Don’t forget to join PPMD on Thursday October 2nd at 1pm EST for a webinar on Vaccinations and Immunity in Individuals with Duchenne and Becker.
Register here: https://parentprojectmd-org.zoom.us/webinar/register/WN__K2_zuxJTlCi_mIn9mmwhQ #/registration

25/09/2025

https://www.capricor.com/investors/news-events/press-releases/detail/326/capricor-therapeutics-provides-regulatory-update-on

FDA and Capricor aligned on endpoints for HOPE-3 pivotal trial HOPE-3 pivotal trial completed; topline data expected mid-Q4 2025 to support BLA…...

18/09/2025

Register for this important webinar about Vaccines and Dystrophinopathy. Thursday, October 2nd at 10am PT https://www.parentprojectmd.org/events/webinar-building-protection-vaccines-and-dystrophinopathy/?fbclid=IwY2xjawM5PElleHRuA2FlbQIxMABicmlkETFKeDFMMzJYNTAzbEV1OTFjAR7plrnzjZEpI_nRQeHsmiULxfp2U5P2vHMouaw_z7IhYJfJWincyrjHh0PBLw_aem_dhwNCKXdEnIPFVxCqygS5Q

Join PPMD for a webinar on October 2, 2025 at 1:00 PM ET to break down the science of vaccines in an easy-to-understand way. We’ll share which vaccines are recommended for people living with dystrophinopathy,...

18/09/2025

The Reauthorization of the Rare Pediatric PRV program has moved to congress. We support The EveryLife Foundation and urge Congress to pass the bill and reauthorize the Rare Pediatric PRV program without delay. You can still make a difference! Support the reauthorization of the PRV program here:

Our fight to reauthorize the Rare Pediatric Disease PRV Program continues. Please join us in urging your members of Congress to reauthorize this critical program by passing the Give Kids a Chance Act of 2025 (H.R. 1262) in the House and a soon-to-be-introduced companion in the Senate.

15/09/2025

Please support adding Duchenne to the RUSP! Today is the deadline to make your voice heard!

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...

10/09/2025

CureDuchenne will host a webinar with Avidity Biosciencies to give update on EXPLORE44-OLE Functional Data on September 18th at 2pm PT. Register here:
https://us02web.zoom.us/webinar/register/WN_Fv8yJJWpSba8d14TOj3USg?fbclid=IwY2xjawMuo0pleHRuA2FlbQIxMABicmlkETFycFhYazlRMlB1Z250Um1CAR5ZXqktXpHMjwgTySWJNq0RDK8XopNmV9YLYuPst19vMCVQy2OxCSYGRuaeGQ_aem_ZNWjKrMkqZ9M1CHeRCHp0w #/registration

Avidity to present functional data to the Duchenne community.

10/09/2025

Avidity Biosciences today announced positive new data from participants treated continuously with del-zota for one year in the EXPLORE44® and EXPLORE44-OLE™ trials. "These data demonstrated reversal of disease progression and unprecedented improvement compared to baseline and natural history across multiple functional measures. Additional data from the EXPLORE44 program will be presented at upcoming scientific congresses."
Full PR here:

-- Unprecedented improvement compared to baseline and natural history in multiple functional measures including Time to Rise from Floor (TTR), 4-Stair Climb (4SC), Performance of Upper Limb (PUL)...

07/09/2025

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...

07/09/2025

Joining with MDA to support World Duchenne Awareness Day.