Center for Duchenne Muscular Dystrophy at UCLA

22/07/2025

Today, Sarepta Therapeutics notified the U.S. Food and Drug Administration of its decision to voluntarily and temporarily pause all shipments of ELEVIDYS (delandistrogene moxeparvovec) for Duchenne muscular dystrophy in the United States, effective close of business Tuesday, July 22, 2025.

“As a patient-centric organization, the decision to voluntarily and temporarily pause shipments of ELEVIDYS was a painful one, as individuals with Duchenne are losing muscle daily and in need of disease-modifying options,” said Doug Ingram, chief executive officer, Sarepta. “It is important for the patients we serve that Sarepta maintains a productive and positive working relationship with FDA, and it became obvious that maintaining that productive working relationship required this temporary suspension while we address any questions that FDA may have and complete the ELEVIDYS label supplement process.”

Sarepta remains committed to transparency and patient safety and will continue to provide timely updates to patients, families, healthcare providers, and the broader Duchenne community as additional information becomes available.
Community letter below.
Full PR here:
https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-voluntary-pause-elevidys

19/07/2025

Sarepta Therapeutics has issued the following statement July 18, 2025:

"Shortly after 2:30 p.m. ET today, Sarepta received an informal request from the U.S. Food and Drug Administration (FDA) to voluntarily halt shipment of ELEVIDYS (delandistrogene moxeparvovec), our gene therapy for Duchenne muscular dystrophy (Duchenne), in the U.S. We first heard of this potential request earlier in the day at the same time the public and our patient communities did, through media reports.

At Sarepta, patient safety and well-being are always our top priority. We are committed to upholding the highest safety standards for all of our therapies. This guides every decision we make, as evidenced by our conservative decision to pause shipments of ELEVIDYS for non-ambulant patients while we work with the FDA to update the label and evaluate the use of an enhanced immunosuppression regimen to mitigate the risk of acute liver failure.

Based on our comprehensive scientific interpretation of the data, which shows no new or changed safety signals in the ambulant patient population, we will continue to ship ELEVIDYS to the ambulant population. We look forward to continued discussions and sharing of information with FDA in order to advance our shared purpose of protecting patient safety and informed access to care."

Full PR and community letter can be found here:
https://www.sarepta.com/

18/07/2025

Medera Inc. a clinical-stage biopharmaceutical company focused on targeting cardiovascular diseases by developing next-generation therapeutics, and its clinical development division Sardocor, together with the University of Kansas Medical Center, today announced the successful treatment of the first patient in the MUSIC-DMD Phase 1b clinical trial. The trial is investigating AAV1.SERCA2a, a one-time gene therapy treatment for cardiomyopathy secondary to Duchenne muscular dystrophy (DMD-CM). PR here:

Landmark MUSIC-DMD Phase 1b trial represents first-in-human gene therapy approach for DMD-associated heart failure...

17/07/2025

Deadline to apply for MDA on the Hill Advocacy is August 11. https://l.facebook.com/l.php?u=https%3A%2F%2Fsway.cloud.microsoft%2FQCbEI4mABs1HHSyr%3Fref%3DLink%26fbclid%3DIwZXh0bgNhZW0CMTAAYnJpZBExY0hhQmlhNmlPSTdIdFVjYQEeqiQMrTTZm5KgsJSlu8B7-BCqffGkA2OCdmu5lR3_VsF0EYAc6aMVbbK_fa8_aem_8mvWli7WYnIUqPFgk26ZZg&h=AT3bSc7Ckx3kuuPKzTLDABvFxXBUcZ9gPfiYIQ7wDH4H787o4ISFeqhXWLx-i83rrMcXFtVp2H7XY5NrkCn_G64TYB3V8mVn0VOqGSFEPlKYa6eOmAj7uiHcl-GxtQBk6MfLw5TUeSK_wkqLaWE&__tn__=-UK-R&c[0]=AT1pCP6fP3BJskOsQBnUpB-l66r7JaQ4oUwE2p5L0sjI62PMjQWn3rP-blUy3HIvwHcIAJr41sqn9yKZgfuYfIAmYkd6cRcgHqRQ_5CoxmsOSHFpY1k5MjDX5OWMS0tSldKyEQdWhgCG9Ed4mzIyuruCAmlSQ7dqaHgPXSiqaV4aiHCO02HAEdlp__Q6dr_Y3MS1rO4rV8plfLnbigVUzvjDeQ

Do you want to join advocates from across the country raising their voices to their members of Congress? If so, join us on November 2-4th for MDA on the Hill. Meeting with your members of Congress and

16/07/2025

Sarepta Therapeutics Announces Strategic Restructuring and Pipeline Prioritization Plan to Maintain Long-term, Sustainable Growth and Provides Update on ELEVIDYS Label.

"Following previously communicated steps being taken to strengthen the safety profile of ELEVIDYS, Sarepta is providing an update on on-going engagement with the U.S. Food and Drug Administration (FDA) regarding the ELEVIDYS (delandistrogene moxeparvovec) label. Consistent with other AAV-delivered gene therapies, the FDA has requested that the label include a black box warning for acute liver injury (ALI) and acute liver failure (ALF). Sarepta agrees with this change, which appears to resolve any material issues with the ambulant portion of the ELEVIDYS label.

Sarepta recently announced that it was pausing shipments of ELEVIDYS for non-ambulant patients while it explored the adoption of additional prophylactic immunosuppression. To that end, Sarepta convened an Expert Committee of neuromuscular specialists, hepatologists, hematologists, and immunologists to review cases of ALF and explore additional immunosuppression regimens. The Committee aligned on an enhanced immunosuppressive regimen with sirolimus for ELEVIDYS in non-ambulant patients. Sarepta will submit the finding of the expert panel and proposed protocol to the FDA imminently and will discuss a proposal to gather data on the regimen in a new cohort (Cohort 8) of the ENDEAVOR study (Study SRP-9001-103) as a pathway to re-establish dosing in the non-ambulant setting. Additionally, Sarepta is assessing real-world data generation opportunities for ambulant patients through investigator-initiated trials."

Full PR here: https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-strategic-restructuring-and

15/07/2025

It’s not too late to register for Team Joseph “What Now? Possibilities in Adulthood with Duchenne” happening tomorrow! Topics include: independence, careers, friendships, mental health, and more. It's a time to learn, connect, and have fun!
If you’re a parent, caregiver or friend, please share this with your loved one (and, you’re welcome, too - read on for details)

Conference Dates: July 16th and 17th, 10am-2pm PT each day
Community Campfire: July 17th, 12:30-2pm PT

This event is FREE and entirely VIRTUAL, ensuring accessibility for all. Parents, caregivers, and community members are invited to join our Community Campfire on the final day.
🔗 Register now: https://whova.com/portal/registration/9lm2WTKUh-QAXV8gFCNe/

14/07/2025

Parent Project Muscular Dystrophy"With all therapies, there are associated side effects and it’s critical that families and individuals who receive gene therapy (or any therapies) understand the signs and symptoms of a potential emergency and when to seek care."

PPMD has created a Post AAV Gene Transfer Therapy Emergency Card as a supplemental resource to the information provided by your/your child’s care team. This card includes critical information for both families and healthcare providers to know what to look for and monitor during potential emergencies after receiving an AAV gene therapy. More info here: https://www.parentprojectmd.org/post-aav-gene-therapy-resources/?fbclid=IwY2xjawLiOMhleHRuA2FlbQIxMQABHrtquh_eBidvKOXph-6vET6jVaRQb_ETkMxaw_t2UpgvqR6kbAlAOzMNEJ19_aem_OB3MhC9-ZtZ0uLnruEp-Tw

As therapeutic options for Duchenne advance, we are seeing continued development of additional AAV gene transfer therapies. There is currently one FDA approved gene therapy, ELEVIDYS, and several more in clinical trials. With all therapies,...

14/07/2025

REGENXBIO Announces Publication of Preclinical Results Demonstrating Functional Benefits of Novel Microdystrophin Construct in RGX-202 Investigational Gene Therapy for Duchenne Muscular Dystrophy. "RGX-202 is the only investigational or approved microdystrophin gene therapy candidate for the treatment of Duchenne muscular dystrophy (Duchenne) that includes the CT domain, a key portion of dystrophin, making it the closest to naturally occurring dystrophin.

"We specifically designed RGX-202 differently from other gene therapies with the goal of providing improved outcomes for patients, and this research further validates the potential therapeutic advantage of adding the CT domain and its importance in preventing the muscle breakdown associated with functional decline in Duchenne," said Olivier Danos, Ph.D., Chief Scientific Officer of REGENXBIO. "The positive interim results we've seen in the Phase I/II AFFINITY DUCHENNE® trial are reinforced by this preclinical research demonstrating how the novel construct of RGX-202 protects against muscle damage and supports the potential for durable, functional benefit for patients."
Press release here: https://ir.regenxbio.com/news-releases/news-release-details/regenxbio-announces-publication-preclinical-results/

11/07/2025

FDA issued Complete Response Letter to Capricor Therapeutics, Inc. Capricor plans to resubmit its BLA to include data from the ongoing Phase 3 HOPE-3 trial in Q3 2025 to continue pursuing the indication for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy.
FDA advised Capricor to request a meeting to determine next steps toward potential approval.

FDA issued Complete Response Letter Capricor plans to resubmit its BLA to include data from the ongoing Phase 3 HOPE-3 trial in Q3 2025 to…...

10/07/2025

CureDuchenne cares will host a free workshop for the Duchenne and Becker communities at The Westin South Coast Plaza on Saturday, August 23 from 8:00 AM – 4:00 PM. Breakfast and lunch will be provided.

09/07/2025

MDA’s new Durable Medical Equipment (DME) Grant Program is opening doors for people living with neuromuscular diseases. This program helps you access essential equipment that supports greater independence, safety, and everyday mobility at home, in school, at work, and at play.

Durable medical equipment can be costly and difficult to access. That’s why MDA is stepping in to ease that burden because everyone deserves the tools that help them live with strength and confidence.

https://www.mda.org/care/resource-list/equipment-assistance?fbclid=IwY2xjawLb3StleHRuA2FlbQIxMQBicmlkETFjSGFCaWE2aU9JN0h0VWNhAR5M-EKI5MLVsXRK1n4Mw2zwDnUEmKTi3LV_YwPKRbf7ov5syGoEKj4h_KPosQ_aem_Q1tVmw5DMHJt5IWLdwUpgw

MDA is the #1 health nonprofit advancing research, care and advocacy for people living with muscular dystrophy, ALS, and related neuromuscular diseases.

27/06/2025

Edgewise Therapeutics Inc., a leading muscle disease biopharmaceutical company, today unveiled positive results in its sevasemten program for Becker and Duchenne muscular dystrophies.
https://investors.edgewisetx.com/news/news-details/2025/Edgewise-Therapeutics-Reports-Positive-Results-on-Sevasemten-Program-for-Becker-and-Duchenne-Muscular-Dystrophies/default.aspx?fbclid=IwY2xjawLKvtxleHRuA2FlbQIxMABicmlkETFya0tXN3Y5QWR3cXJnaW10AR4mvJFMP108f55sFPdiLLirOsdUrXUYmy1nXu8zwvHOkMeaZj7SfSnkvFjesw_aem_5kXhjnFJ0hV1w3-5WwZMDA

New open label data in Becker demonstrated sustained disease stabilization up to three years, reinforcing prior clinical findings Ongoing pivotal trial and FDA Type C meeting provide clear path to potential sevasemten registration as the first ever therapy for Becker Encouraging Phase 2 observations...