CureDuchenne

12/17/2025

Watch the webinar with Dr. Doug Kerr, Chief Medical Officer at Dyne Therapeutics.

Dr. Kerr will share topline results from the DELIVER clinical trial, along with new long-term data showing sustained benefits out to 24 months.

DELIVER is Dyne's global, randomized, placebo-controlled, double-blind, Phase 1/2 trial that evaluated the safety, tolerability and efficacy of zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) in individuals with Duchenne muscular dystrophy who have mutations in the DMD gene that are amenable to exon 51 skipping.

WATCH WEBINAR: https://cureduchenne.org/general/dyne-webinar-positive-topline-results-from-deliver-trial-in-exon-51-dmd/

12/17/2025

FUTURES 2026 is around the corner, and we can’t wait!!
Registration is now open for Futures 2026, May 21-24 💙

We are excited for another amazing year with our CureDuchenne families! This year we will be welcoming you in Orlando 🤩

Register today at https://web.cvent.com/event/ee9e7402-2c59-420f-9cf9-5731cf20d4d9/summary

12/16/2025

BREAKING NEWS: CureDuchenne applauds the U.S. Department of Health and Human Services (HHS) for adding Duchenne muscular dystrophy to the Recommended Uniform Screening Panel (RUSP) for newborn screening.

We also extend our congratulations to Parent Project Muscular Dystrophy (PPMD) for leading the nomination and to Muscular Dystrophy Association (MDA), the co-sponsor. Several pilot newborn screening programs—including one supported by CureDuchenne—were instrumental in showing that Duchenne screening can be implemented effectively and that the diagnostic tools are working.

This forward-thinking and evidence-based decision marks a pivotal step, as earlier diagnosis offers families a better chance to preserve function, avoid delays, and begin care and treatment when it may be most effective. This milestone comes at a critical time: three Duchenne research programs that received early funding from CureDuchenne are expected to apply for FDA approval in the coming year. As the pipeline of potential treatments grows, identifying individuals as early as possible becomes even more essential.

We’re grateful to the advocates, families, scientists, and clinicians who made this moment possible. As newborn screening moves toward implementation in states, CureDuchenne will continue to support families, clinicians, and partners every step of the way.

Learn more here: https://cureduchenne.org/research/breaking-news-duchenne-muscular-dystrophy-has-officially-been-added-to-the-recommended-uniform-screening-panel-rusp-for-newborn-screening-by-the-u-s-department-of-health-and-human-services-this-m/

12/16/2025

Spread some real holiday joy—donate today to give Deklan and so many others more than just time 💙

Your gift right now fuels the research that moves us closer to treatments—and ultimately, a cure.
You can be the reason a cure becomes reality 💫

Donate today at https://cureduchenne.org/donate/

CureDuchenne’s mission - cure Duchenne muscular dystrophy (DMD). Our venture philanthropy model funds research, early diagnosis, treatment

12/10/2025

Raise Your Voice. Rally Your Community. Start a fundraiser today. 💙

Every day, families like yours are rewriting what’s possible.

By showing up, speaking out, and sharing your stories, you're helping drive science forward and bringing us closer to the day when no child has to live with Duchenne.

Now, we’re inviting you to take that next step:
Start your own personal fundraising page and join CureDuchenne’s mission to fund the research that will change lives.

But this isn’t just about supporting our work, this is about uniting as one community with one shared goal: to cure Duchenne for every child, everywhere.

Whether you're celebrating your child's birthday, honoring a milestone, or raising awareness, your voice matters. And your story can move people to action.

Together, we can bring more therapies to trial, more care to families, and more hope to everyone affected by Duchenne.

Setting up your page is quick and easy. You’ll get your own custom link to share with friends and family. Every dollar raised helps accelerate research, improve care, and change what's possible.

👉 Use the link below to create your own fundraiser!

https://igfn.us/vf/25EOY

12/09/2025

Art meets wine, with purpose. 🎨🍷

Napa native Blake Barrios is rewriting the rules with Rebellium, a visionary wine-art hybrid where each hand-painted magnum becomes a story, a statement, and a vehicle for change.

From celebrity collaborations to powerful partnerships with nonprofits like , Barrios merges artistry and philanthropy in every bottle.

Read more about how became a cause for good: https://napavalleylife.com/digital-edition/

12/09/2025

Satellos Biosciences has received Investigational New Drug (IND) clearance from the FDA, as well as other global regulatory agencies, to conduct a 3-month placebo-controlled Phase 2 study of SAT-3247 in ambulatory individuals with Duchenne.

SAT-3247 is an oral, small molecule drug aiming to regenerate skeletal muscle that is lost in Duchenne. Satellos expects the first participant to be enrolled by the end of the year, and will report interim data in Q2 2026.

Read the press release HERE

Satellos receives clearance by FDA to initiate Phase 2 study in Duchenne

12/08/2025

What an incredible night at our CureDuchenne Family Dinner Session in Phoenix on Saturday night! The room was full of connection, conversation, and community — everything we hope for when families come together.

A highlight of the weekend? Our amazing PT had a chance to share with Mr. and Mrs. Claus all about Duchenne… while climbing Camelback Mountain! Only in Phoenix do you get advocacy, sunshine, and Santa on a hike all in one day.

We’re also deeply thankful for our sponsors, whose support makes these meaningful gatherings possible for our families.

Grateful for everyone who joined us, the important conversations, and the continued dedication of this strong and caring community.

12/08/2025

As an early investor in Dyne Therapeutics, we are pleased to share their exciting news that skipping exon 51 in the dystrophin gene with z-rostudirsen in the registrational expansion cohort met the primary endpoint--a statistically significant increase in dystrophin at 6 months—and was also associated with functional improvements in multiple measurements.

The details:
The DELIVER trial was a randomized, placebo-controlled, double-blind, Phase 1/2 trial that evaluated the safety, tolerability, and efficacy of zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) in individuals with Duchenne who are amenable to exon 51 skipping. DELIVER consisted of two parts:

⏺️Part 1 was a multiple ascending dose (MAD) study, designed to identify the appropriate dose of z-rostudirsen in small numbers of participants

⏺️Part 2 was the Registrational Expansion Cohort (REC), designed to test the selected dose of z-rostudirsen in a larger number of participants to evaluate safety and efficacy. Trial participants who have completed the MAD and the REC continue to receive z-rostudirsen as part of the open label extension portion of the trial.

Along with a favorable safety and tolerability profile, positive topline results from DELIVER Registrational Expansion Cohort (REC) of 32 participants include:

⏺️Dystrophin: The REC met its primary endpoint, demonstrating a statistically significant increase in dystrophin protein expression of 5.46% (muscle content-adjusted) at 6 months. This replicated the same 7-fold change from baseline previously observed.

⏺️Function: Improvement relative to placebo was seen across all six prespecified endpoints at six months. The six endpoints cover upper and lower limb function, as well as lung function.

In addition, new positive long-term results from DELIVER showed sustained functional improvement across multiple endpoints out to 24 months in each of the six endpoints for participants in the MAD cohort.

What comes next?
Dyne plans to submit a potential Biologics License Application (BLA) for U.S. Accelerated Approval in Q2 2026. If the FDA issues priority review and all goes well, the timing could point towards a potential commercial launch in Q1 2027.
Dyne also plans to initiate a global Phase 3 clinical trial of z-rostudirsen in Q2 2026, to support approvals outside of the US.

Click here to read more and see letter to the community: https://cureduchenne.org/research/dyne-announces-positive-topline-results-from-phase-1-2-trial-of-z-rostudirsen-in-duchenne-amenable-to-skipping-exon-51/

Dyne Therapeutics

12/03/2025

Capricor Therapeutics, Inc., which received $1.5M from CureDuchenne in 2015, announced today that HOPE-3 met its primary endpoint and a key cardiac secondary endpoint, with all Type 1 error–controlled secondary endpoints also achieving statistical significance.

HOPE-3 is a randomized, double-blind, placebo-controlled, Phase 3 clinical trial evaluating Deramiocel in boys and young men with Duchenne muscular dystrophy. The study randomized 106 participants across 20 leading U.S. clinical sites. Participants received intravenous Deramiocel at 150 million cells per infusion or placebo every three months for a 12-month period.

The average age of participants was approximately 15 years, and all were on a stable corticosteroid regimen throughout the study.

Baseline demographics were well balanced between treatment arms, approximately 90 percent were receiving cardiac medications at baseline, and over 75 percent had a clinical diagnosis of cardiomyopathy. Deramiocel maintained a favorable safety and tolerability profile consistent with prior clinical experience.

Read more here: https://cureduchenne.org/research/capricor-theraputicsannounces-positive-topline-results-from-pivotal-phase-3-hope-3-study-of-deramiocel-in-duchenne-muscular-dystrophy/

12/02/2025

Today is the day to give. 💙
On this , your support brings us closer to a cure for guys like Rocco, Josh, and Deklan.

Swipe to meet these incredible young men and see the impact your gift can make.

Every dollar helps fund research, expand access to care, and create a future with more birthdays and more breakthroughs.

Together, we’re changing what’s possible for everyone living with Duchenne.

Donate at https://fundraise.givesmart.com/e/3qoxXw?vid=1n97m7