Center for Duchenne Muscular Dystrophy at UCLA

26/11/2025

Feeling gratitude for all of the people and organizations who have supported the CDMD at UCLA thus far and thankful for the doctors, scientists, and nurses on our team. And especially grateful to the patients and families we serve. Wishing everyone a Peaceful Holiday Weekend.

25/11/2025

Sarepta Announces Approval to Begin 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-approval-begin-endeavor-cohort-8-evaluate

24/11/2025

Grateful for Dr. Nelson and Dr Miceli, our CDMD Co Directors and 'Cellular Pioneers,' featured in Rare Revolution Magazine.

Rare Revolution - 27/08/2025

24/11/2025

On November 19, Avidity Biosciences announced its Managed Access Program (MAP) for investigational therapy delpacibart zotadirsen (del-zota) for eligible people with Duchenne muscular dystrophy mutations amenable to exon 44 skipping (DMD44) in the United States.
"We recognize the considerable needs facing the DMD44 community given there are no approved exon skipping therapies for this disease as well as the potential of del-zota based on the unprecedented data shown in our clinical studies," said Sarah Boyce, President and Chief Executive Officer of Avidity. "We are pleased to open this MAP to enable a compliant approach to providing del-zota to eligible patients as quickly as possible."

Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced...

16/11/2025

Community Letter from Sarepta regarding the label change for Elevidys:

November 16, 2025
Dear Duchenne community,
Recently, we shared the announcement that the prescribing information (also known as “the label”) for ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for Duchenne muscular dystrophy (DMD) has been updated. With this letter, we want to share what has changed, provide the MedicationGuide, answer questions about what it means for families and patients interested in pursuing treatment, and reaffirm our commitment to ensuring patient safety, which is our highest priority.

What is a label change?
The prescribing information (PI) label is a living document that provides instructions and essential scientific information to healthcare professionals for the safe and effective use of a medicine. Often times, especially in rare disease, as real-world experience with a prescribed product grows post- approval, updates to the label are made to reflect new learnings around patients’ experiences with the therapy, including those related to adverse events.
Sarepta initiated the labeling update process for ELEVIDYS following the passing of a non-ambulatory patient from acute liver failure following treatment with ELEVIDYS, and has been working with FDA over the last several months to make updates to the labeling.
The revised labeling can be accessed PILINK as well as the MedicationGuide.

What changed with the ELEVIDYS label?
As previously shared, we voluntarily suspended commercial shipments of ELEVIDYS for non-ambulatory patients in June, while we pursued plans for an additional study of an enhanced immunosuppressive regimen in non-ambulatory patients.
At this time, the FDA review of the updated label is complete. Here we share key updates to the ELEVIDYS label:
• A boxed warning for the risk of acute serious liver injury (ALI) and acute liver failure (ALF). A boxed warning is meant by FDA to highlight certain contraindications or serious warnings and to call prominent attention to healthcare providers about potential adverse reactions and safe use instructions.
• Removal of language regarding the use of ELEVIDYS for non-ambulatory patients. These measures aim to ensure the safe administration of ELEVIDYS in ambulatory patients while equipping healthcare providers, patients, and families with the scientific information needed to support informed, individualized treatment discussions.
• Expanded guidance for prescribers, including a modified pre- and post-infusion oral corticosteroids regimen and enhanced monitoring recommendations on a weekly basis for 3 months post-infusion to help ensure patients receiving ELEVIDYS have a standardized pre- and post-infusion treatment and monitoring experience.
• A new warning in the Warnings & Precautions section regarding increased susceptibility for serious infections due to immunosuppression.
Our commitment to non-ambulatory Duchenne patients remains unwavering. This label update allows us the time to study the use of enhanced immunosuppression therapy in non-ambulatory patients such that we will be able to have a more informed, evidence-based discussion regarding the path to future access to ELEVIDYS by non-ambulatory patients.
We understand the urgency this group of patients and their families feel; we feel it, too. We will work diligently with regulators and healthcare professionals and, most importantly, you, the Duchenne community, to ensure that non-ambulatory patients have equal access to informed decision-making about their care and the benefit-risk profile of innovative treatment options and will continue to support the entire Duchenne community.
We encourage families to read the Medication Guide and to talk to their doctor for a full understanding of the risks and benefits on an individual patient level. https://www.elevidys.com/med-guide
Sincerely,
Wendy Erler
Senior Vice President, Patient Affairs

14/11/2025

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

14/11/2025

Nov. 14, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced an update to the prescribing information for ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for Duchenne muscular dystrophy (DMD).

Full PR here: https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-announces-fdas-approval-updated-elevidys-prescribing

12/11/2025

“We are entering one of the most pivotal periods in Capricor’s history as we approach the topline readout from our HOPE-3 Phase 3 trial of Deramiocel for the treatment of Duchenne muscular dystrophy,” said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “Deramiocel was developed to address the cardiomyopathy that ultimately claims the lives of nearly all patients with Duchenne, and our mission has never been clearer: to bring forward the first therapy specifically designed to target this life-limiting aspect of the disease. Over the past decade, we have generated compelling and statistically significant data showing durable improvements in both cardiac and skeletal muscle function. With our commercial-ready manufacturing facility in place and our Pre-License Inspection completed, we believe we are well positioned for potential approval and launch. We remain confident in the strength, consistency, and reproducibility of our science and fully focused on advancing Deramiocel toward approval and commercialization, with the broader goal of delivering meaningful and lasting value to patients, families, and shareholders.”
Full PR here:

Topline results from pivotal HOPE-3 Phase 3 study (n=105) of Deramiocel for the treatment of Duchenne muscular dystrophy expected in the coming…...

11/11/2025

Sarepta Community Letter

November 10, 2025

Dear Duchenne Community,

After the Sarepta community letter this week, we heard a lot of feedback from you, the community, about your disappointment in the amount of information we shared, particularly related to the global PMO study, ESSENCE (the confirmatory trial of two Sarepta therapies, VYONDYS 53 and AMONDYS 45). Your feedback is appreciated and critical for us to receive.

We want to take a moment to reiterate our commitment to you and speak more about how and when we share information—particularly around data updates, milestones, and other developments related to our medicines. We understand how deeply personal and important this information is to patients, families, and advocates. Your trust and engagement are central to everything we do. I am writing to you today to reaffirm our deep commitment to you, and that we remain steadfast in our commitment to advance treatments for the Duchenne community.

The ESSENCE Study & Next Steps

As we announced publicly, we are still analyzing the full results from the ESSENCE study. We are also preparing to meet with the FDA to discuss the results and the real-world data in detail and the potential for traditional approval.

We want to provide some context for the way in which this information was communicated this week to the community, to investors, and to the public. As a publicly traded company, we are required to share material updates publicly and in our securities filings. While the Securities and Exchange Commission governs how we make those communications, the FDA has its own requirements – and limits – on the sharing of clinical trial and real-world data with patients and healthcare professionals. These requirements are designed to protect patients and to ensure the scientific rigor necessary to develop safe and effective treatments.

As a result, we must carefully navigate what data and information we can and must share and when and how we share it, which was why we communicated some information in our earnings announcement and other information in our community letter this week. We know that these strict guardrails can be frustrating not only for our team members but also for the Duchenne community. Please know that while we must adhere to these requirements in the way we share updates publicly, we remain steadfastly committed to close collaboration with the community in the development of much-needed treatments for Duchenne.

When we have fully analyzed the data from ESSENCE, and discussed them together with the FDA, we will move forward with transparency and compliantly to share any new learnings from these data and our next steps with the community.

Looking Ahead Together

We are deeply grateful for your continued support of our research work. Your advocacy, participation in clinical trials, and willingness to share your stories are invaluable contributions to the advancement of rare disease research. Patients, families, and advocates are at the heart of everything we do. We understand that rare diseases present unique challenges—not only for those living with these conditions but also for the families and caregivers who provide daily support. Your experiences, insights, and engagement inspire our research efforts and remind us daily of the importance of our mission.

Together, we are working towards a future where better treatments and, ultimately, cures may be possible. We remain committed to engaging with you in meaningful ways while meeting our obligations to regulators, and we will continue to share information as openly as possible. Thank you for your partnership, your patience, and your dedication.

With gratitude and respect,

Wendy

ACTIVE/203349535.1

10/11/2025

Amazing accomplishment! Thank you to all the PAAC members for your service to the B/DMD Community and to PPMD for creating the incredible PAAC program!
"PPMD’s Adult Advisory Committee (PAAC) recently expanded with 19 inspiring new members, bringing the committee to its largest size ever—73 members strong! These incredible individuals bring diverse academic backgrounds ranging from marketing and communication to engineering, cognitive science, and even a PhD in disability studies. They also speak multiple languages including Spanish, Russian, French, and Hindi, helping to grow PAAC’s community reach."
https://www.parentprojectmd.org/welcoming-the-new-members-of-paac-for-fall-2025/?fbclid=IwY2xjawN_HdxleHRuA2FlbQIxMQBzcnRjBmFwcF9pZBAyMjIwMzkxNzg4MjAwODkyAAEeTMAl4Qbnn_d5wIN_xjrmwRYZXWCP3Dkf1FB-ToQkenvF0w0trbrrZ7pDGUk_aem_1LlCoD742q3mBEQufi9Kwg

PPMD’s Adult Advisory Committee (PAAC) recently expanded with 19 inspiring new members, bringing the committee to its largest size ever—73 members strong. These incredible individuals bring diverse academic backgrounds ranging from marketing and communication to...