CureDuchenne

11/16/2025

Boston showed UP today! Our CureDuchenne workshop was filled with incredible families, heartfelt conversations, and four truly inspiring adults living with Duchenne who shared their journeys with so much honesty and strength. The UMASS Duchenne Clinic team brought amazing insight and compassion, making the day even more meaningful. And a huge, heartfelt thank-you to our sponsors—you help us bring this community together, and we couldn’t do it without you.

11/15/2025

Watch the webinar:

Watch webinar HERE

11/15/2025

The FDA has approved the new labeling information Sarepta provided for Elevidys, Sarepta’s gene therapy for Duchenne. This update comes after reports of fatal acute liver failure in non-ambulatory patients, and a review of all of the available safety data.

The updates include:
A Boxed Warning for the risk of serious liver injury and acute liver failure, including fatal outcomes-
- Limiting the indication to ambulatory individuals (aged 4 years and older) only. The non-ambulatory indication has been removed.
- Various additions to help guide prescriber’s clinical decision making, including modified pre- and post- infusion corticosteroid regimen, and enhanced monitoring recommendations for 3 months post-infusion
- A new Warnings & Precautions regarding increased susceptibility to serious infections
- A new Medication guide for patients and caregivers

The FDA has also asked Sarepta to conduct a postmarketing observational study in 200 individuals and follow them for at least a year after treatment, with periodic liver assessments to further assess the risks.

For non-ambulatory individuals, Sarepta has indicated they plan to work with the FDA to commence a study using an enhanced immunosuppressive regimen using sirolimus, in conjunction with Elevidys dosing.

Read more here:

This update comes after reports of fatal acute liver failure in non-ambulatory patients, and a review of all of the available safety data.

11/15/2025

We’re thrilled to be part of the 30th year of Champions for Charity® at Americana Manhasset! ☺️

From December 4 - 6, 2025, your holiday shopping can help support our cause. Simply register for your champion number and present it at the time of each purchase so 25% of your designated full-price, pre-tax purchases will be donated back to us.

Visit championsforcharity.org for details.

11/14/2025

We congratulate C-PATH (Critical Path Institute) on their success in getting the FDA to qualify a new biomarker-- glutamate dehydrogenase (GLDH)-- to monitor drug-induced liver injury in clinical trials.

This development is important in Duchenne and Becker because other biomarkers typically used to monitor liver injury (such as alanine aminotransferase, or ALT) can already be elevated in individuals with muscle injury, making interpretation of results more difficult. GLDH is a more specific marker of liver injury, and its inclusion in clinical trials in individuals with muscle injury represents an improvement in liver safety monitoring.

As a member and data contributor to C-PATH’s Duchenne Regulatory Science Consortium, we are so pleased to have been part of this important milestone.

As a member and data contributor to C-PATH’s Duchenne Regulatory Science Consortium, we are so pleased to have been part of this important milestone.

11/14/2025

On Saturday, November 22, locals in Ellworth, ME, will get to see Maine in a whole new way, thanks to an incredible film made by Evan Procko.

Evan, who lives with Duchenne muscular dystrophy, created this beautiful piece to support CureDuchenne and share his unique perspective. His talent, heart, and storytelling are inspiring, and we’re so grateful for the way he’s using his voice to raise awareness and funds for research.

Check out the story and details about the screening here:
🔗 www.wabi.tv/.../maine-filmmaker-living-with...

Thank you, Evan, for sharing your art and your voice! 💙

On Saturday, November 22, those who head to The Grand Theater in Ellsworth will get a chance to see the state of Maine in a way they’ve likely never seen before.

Story: https://www.wabi.tv/2025/11/12/maine-filmmaker-living-with-muscular-dystrophy-premiere-nature-film-proceeds-benefiting-research/

11/14/2025

Thank you to the Terry family for your incredible support in our mission to cure Duchenne! 💫
Together, we’re making a difference for everyone affected by Duchenne muscular dystrophy.
https://www.youtube.com/watch?v=tahEL3cjD3o
Join our community of supporters — secure your sponsorship today at https://cureduchennegolfshootout.org/ 💪
Together, we will cure Duchenne💙

Event date: September 11-12, 2026. We hope to see you there!

The CureDuchenne Golf Shootout, started by NHL All-Star Ryan Getzlaf and his wife Paige and now hosted by Troy Terry of the Anaheim Ducks and his wife Daniel...

11/13/2025

When you love someone with , doing something becomes its own kind of healing. This free webinar brings together inspiring families who turned their passion into purpose—and offers practical tools so you can do the same.

Whether your event is big or small, it makes a real impact.
You’ll also learn how can help you attend the FUTURES 2026 conference and connect deeper with the community.

📅 Date: Wednesday, December 10, 2025
🕐 Time: 10 AM Pacific Time | 1 PM Eastern Time
📍 Zoom Registration Link: https://us02web.zoom.us/webinar/register/WN_u6nR4-ubQxKkpl9HTnjTDw #/registration

11/10/2025

Chicago showed up this weekend! What an amazing turnout for our CureDuchenne Dinner Session — great food, great conversations, and lots of new connections (and exchanged numbers!). 💙 Thank you to everyone who came out to be part of this special night. Thanks to our sponsors for their support of these beautiful events!

11/06/2025

As an early investor in Entrada Therapeutics, we are pleased to share their progress on their exon-skipping programs in Duchenne.

For Exon 44: Entrada completed enrollment for the first cohort of the ex-US ELEVATE-44-201 study for those amenable to skipping exon 44, and are on track to report data in Q2 2026. They expect to initiate a Phase 1b study in the US for ambulatory and non-ambulatory adults with Duchenne in 2026.

For Exon 45: Entrada has dosed the first participant in the ELEVATE-45-201 study for those amenable to skipping exon 45, and will report data from the first cohort in mid-2026.

For Exon 50: Entrada filed for regulatory authorization in the U.K. for the ELEVATE-50-201 study to begin the study there by the end of 2026.

Link to press release:
https://ir.entradatx.com/news-releases/news-release-details/entrada-therapeutics-reports-third-quarter-2025-financial

11/04/2025

Solid Biosciences today reported an update on the progress of the INSPIRE DUCHENNE open label, Phase I/II clinical trial with SGT-003 and their planned regulatory discussions in 2026.

23 Pediatric participants have been dosed (1E14vg/kg) so far in the INSPIRE DUCHENNE trial (data cutoff 31st October). SGT-003 was generally well tolerated, there were no cases of drug-induced liver injury observed (n = 23) and day-90 biopsy data from 10 treated participants (ages 5-10) showed that all responded to treatment with mean muscle microdystrophin expression of 58% by both western blot and mass spectrometry.

The company expects to have dosed 30 participants by early 2026, and plans to meet with the FDA to discuss a potential registrational pathway in the first half of 2026.

Solid Bio also reported interim clinical data that included correlations observed between SGT-003 microdystrophin therapy and improvements in several biomarkers of muscle integrity, including serum creatine kinase, lactate dehydrogenase, aspartate aminotransferase and embryonic myosin heavy chain, that suggested an effect of treatment with SGT-003.

The company plans to host a number of educational events/webinars in the coming weeks to communicate todays results to the broader Duchenne community.

Read the press release:
https://cureduchenne.org/research/solid-plans-to-seek-fda-guidance-in-2026-for-duchenne-gene-therapy-candidate-sgt-003-s/

Solid Biosciences today reported an update on the progress of the INSPIRE DUCHENNE open label, Phase I/II clinical trial with SGT-003 and their planned regulatory discussions in 2026.

11/04/2025

Sarepta has completed the ESSENCE study, which is a confirmatory trial aimed at supporting full FDA approval of the exon-skipping PMOs Amondys 45 and Vyondys 53. The study did reinforce the favorable safety profile of the therapies, but did not achieve statistical significance on the primary endpoint (4-step ascend velocity at 96 weeks).

What comes next? Sarepta says it plans to discuss with the FDA a path from accelerated approval towards full approval, utilizing this data as well as real world evidence of these therapeutic approaches collected over multiple years.

Read the press release and letter to the community:
https://cureduchenne.org/general/sareptas-amondys-45-and-vyondys-53-confirmatory-trial-fails-to-achieve-statistical-significance-on-primary-endpoint-v2/