Center for Duchenne Muscular Dystrophy at UCLA

01/23/2026

Please join PPMD for a community webinar with Dyne Therapeutics on Wednesday, January 28th at 1:00 PM ET. The webinar will feature a presentation with Dr. Doug Kerr, Dyne’s Chief Medical Officer, who will share topline results from the DELIVER clinical trial, along with new long-term data showing sustained benefits out to 24 months.
Register here:
https://www.parentprojectmd.org/events/webinar-dyne-therapeutics-topline-results-from-phase-1-2-deliver-trial/?fbclid=IwY2xjawPg2uZleHRuA2FlbQIxMQBzcnRjBmFwcF9pZBAyMjIwMzkxNzg4MjAwODkyAAEe_hTphWSuVSmb2W7fevlzjnW_pdNzhfGFPkbEei5dkdZ936-Y-Fgd2668ORo_aem_AOWQutdEUJOkIv295I-DTw

Please join us for a community webinar with Dyne Therapeutics on Wednesday, January 28th at 1:00 PM ET. The webinar will feature a presentation with Dr. Doug Kerr, Dyne’s Chief Medical Officer, who will share...

01/23/2026

On Monday Jan. 26 at 8:30am Eastern Time, Sarepta will share 3 year topline results from the Embark Phase 3 Study of Elevidys.

"Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that on Monday, Jan. 26, 2026, at 8:30 am Eastern Time, the Company will host a webcast and conference call to present 3-year topline functional results from patients treated in Part 1 of EMBARK (Study 9001-301), the global, randomized placebo-controlled Phase 3 study evaluating ELEVIDYS (delandistrogene moxeparvovec-rokl) in ambulatory individuals with Duchenne muscular dystrophy who were aged four to seven at time of treatment."
Register Full PR here: https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-report-3-year-topline-data-embark-study

01/21/2026

Action Alert from PPMD! Urge Congress to support FY 2026 Duchenne funding. Click link and send emails to your representatives:
https://engage.parentprojectmd.org/component/sbxactions/36?fbclid=IwY2xjawPeNLBleHRuA2FlbQIxMABicmlkETF2ZTdraW9xRzBudXMzaEtkc3J0YwZhcHBfaWQQMjIyMDM5MTc4ODIwMDg5MgABHsGOjZcINI3JmX9G85lQVH7vWYtqHl5MNmLuWeiZspF4uZ5Dh1uRJ43YoLRg_aem_DT61k9xlaEh4GFzNC0GQbg

Send a message to your Members of Congress urging them to support our priorities

01/21/2026

“We are actively engaging with the FDA in order to facilitate an efficient review of the HOPE-3 data that directly address the issues raised in the CRL we received in July 2025. We were pleased that the FDA requested the HOPE-3 clinical study report, as this is an expected and appropriate next step following their initial review of the topline data,” said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “The HOPE-3 results demonstrated statistically significant and clinically meaningful improvements in both skeletal muscle and cardiac function—key drivers of disease progression and long-term outcomes in Duchenne. These findings build on more than a decade of consistent clinical evidence and reinforce our confidence in Deramiocel’s potential. Our near-term priority is to address the FDA’s request and continue working collaboratively so that patients with late-stage DMD, who currently have very limited treatment options, may gain access to Deramiocel as soon as possible.”

Full PR Here:

FDA has requested the HOPE-3 clinical study report (CSR) as part of the BLA review process Company expects to submit updates to the BLA in…...

01/13/2026

Sharing From Everylife Foundation: "2026 offers us unique opportunities to push the Give Kids a Chance Act over the finish line and ensure that our children receive lifesaving treatments."

In the short term, the bill has two paths forward for passage:
- Another unanimous consent vote attempt; or
- Its addition to a broader healthcare or funding legislative package, which may be considered at the end of January or in the months following. "

⁉️ What's next for the Give Kids a Chance Act?

The rare disease community starts the year with renewed efforts to reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) Program. 2026 offers us unique opportunities to push the Give Kids a Chance Act over the finish line and ensure that our children receive lifesaving treatments.

In the short term, the bill has two paths forward for passage:
- Another unanimous consent vote attempt; or
- Its addition to a broader healthcare or funding legislative package, which may be considered at the end of January or in the months following.

➡️ To read more about PRV updates, visit our page: https://everylifefoundation.org/rare-disease-community-renews-efforts-to-reauthorize-the-rare-pediatric-disease-priority-review-voucher/

➡️ Tell your Senator to support the Give Kids a Chance Act: https://everylifefoundation.quorum.us/campaign/110385/

01/13/2026

January 13, 2026
Solid Biosciences Provides 2026 Outlook Underscoring Neuromuscular and Cardiac Pipeline Momentum and Expanded Access to Next-Generation Capsid AAV-SLB101

- Duchenne: Dosed 33 participants in the Phase 1/2 INSPIRE DUCHENNE clinical trial as of January 9, 2026; SGT-003 continues to be generally well tolerated using a steroid-only prophylactic immunomodulation regimen -

- Duchenne: First participant enrolled in IMPACT DUCHENNE, a Phase 3 randomized, double-blind, placebo-controlled, ex-U.S. clinical trial, with dosing expected in Q1 2026 -

Community letter Here: https://www.solidbio.com/letter-to-the-duchenne-community-jan2026/

Full pr here: https://investors.solidbio.com/news-releases/news-release-details/solid-biosciences-provides-2026-outlook-underscoring

01/13/2026

REGENXBIO announced new, positive 18-month functional data from patients treated with the pivotal dose in the Phase I/II portion of the AFFINITY DUCHENNE® trial (n=4). All patients exceeded expected disease trajectory on the North Star Ambulatory Assessment (NSAA) using the established cTAP disease progression model. RGX-202 recipients improved an average of 7.4 points compared to cTAP. These same patients improved an average of 6.6 points compared to cTAP at 12 months post-treatment. The Company plans to share additional Phase I/II safety, biomarker, and functional data at the MDA Clinical and Scientific Conference in March 2026.
Clinical Trial and Regulatory Milestones

REGENXBIO expects to share pivotal topline data in early Q2 2026 and submit a Biologics License Application (BLA) under the accelerated approval pathway in mid-2026. Following the completion of enrollment in the pivotal trial (n=30) in October 2025, the Company continues to enroll in the confirmatory trial and expects to have majority of this trial enrolled at the time of BLA filing.
Regulatory interactions with the FDA and European Medical Association (EMA) are planned for 1H 2026, supporting the global expansion of the AFFINITY DUCHENNE® trial.
Full PR here:

New Phase I/II RGX-202 functional data demonstrates long-term, durable treatment effect at pivotal dose at 18 months Robust patient enrollment in confirmatory...

01/13/2026

Are you a parent or caregiver navigating the complexities of life with Duchenne muscular dystrophy (DMD)? Are you seeking a safe, supportive space to share your experiences, ask questions, and build meaningful connections?
PPMD invites you to join the upcoming season of Lighthouse Workshops with Rachel Callander - a nurturing, compassionate container dedicated exclusively to parents, caregivers, and carrier mothers who understand your unique journey.
Register for this season by January 30th. More info here:

PPMD Lighthouse is a wellness and mental health initiative that encourages conversation, promotes connectivity, and supports overall wellbeing. It is our hope that PPMD Lighthouse Workshops will provide a safe space to build stronger connections and deep...

01/12/2026

The Muscular Dystrophy Association (MDA) College Scholarship program provides students living with neuromuscular disease with support as they pursue their academic dreams. The 2026 application cycle is open. Application deadline is March 15, 2026.

The MDA College Scholarship program recognizes students who are making an impact in the neuromuscular community and demonstrate leadership qualities.

MDA grants merit-based college scholarships each year at up to $5,000 each. The application period opens early each calendar year, and applicants receive response notifications in early summer.
More here:

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

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.