CureDuchenne

02/27/2026

This Rare Disease Day, we honor the power of connection, community, and hope. 💙

Robert and Trude Steen — the inspiring parents of Mats Steen — remind us that rare does not mean alone.

In sharing their story, they shine a light on what so many families experience: the challenges, the isolation, and the deep need for community. That’s why CureDuchenne exists. We bring families together. We accelerate research. We create spaces where no one has to navigate Duchenne alone.

Hope grows when stories are shared. Strength grows when community shows up. And progress happens when we invest boldly in a better future.

Today, and every day, we stand with the Duchenne community and the 300 million people worldwide living with a rare disease.

💙 You are not alone.

02/27/2026

On , we stand with the 300 + million people around the world living with a rare condition — including those affected by Duchenne muscular dystrophy. 💙

Rare diseases often go unseen and underfunded, yet the need for awareness, research, and meaningful investment has never been greater.

Duchenne is a rare, progressive muscle-wasting disease that impacts families every day — and while science is essential, bold and strategic funding is what turns hope into real progress. That’s why our CEO, Debra Miller, is calling for more than good science — she’s calling for bold investment to accelerate research and bring treatments closer to patients.

👉 Read her full perspective:
🔗 https://www.futureofpersonalhealth.com/rare-diseases/rare-disease-research-needs-more-than-good-science-it-needs-bold-investment/

Today and every day, we raise our voices, share our stories, and work toward a future where rare is no longer overlooked. 💫

Scientific breakthroughs alone aren’t enough. Bold, strategic investment is what turns hope into real progress.

02/27/2026

📍 Baltimore Families — This One's for You!
If Duchenne is part of your story, you won't want to miss the CureDuchenne Workshop in Baltimore!

Connect with experts, hear from others walking this journey, and leave with information and hope you can use.

Featured Speakers:
🎤 Jake Marrazzo — Hear Jake share his Duchenne story in his own words, his own way. This is one you won't want to miss.

🩺 Dr. Andreas Barth, MD, PhD — Electrophysiology Cardiology, Johns Hopkins — bringing world-class cardiac expertise straight to our community.

🩺Doris G. Leung, MD, PhD, Director, Center for Genetic
Muscle Disorders at Kennedy Krieger Institute

Whether you're newly diagnosed or have been on this road for years, this workshop is your space to learn, to ask questions, and to connect with a community that truly understands.

🔗 Register now: https://cureduchenne.pro/Baltimore
📅 Date: March 28, 2026 | 8 AM – 4 PM
📍 Location: Marriott Inner Harbor at Camden Yards

Tag a Duchenne family who needs to see this! 👇

02/26/2026

Today, as Doug Ingram announces his upcoming retirement, we thank him sincerely for his leadership and commitment to advancing treatments for people living with Duchenne.

During his tenure as CEO of Sarepta Therapeutics, the field moved forward in meaningful ways, and Sarepta brought new therapies to families who had waited decades for options. Progress in Duchenne takes courage, urgency, and collaboration, and we recognize the important role that Doug and Sarepta have played in accelerating treatments.

As a community, we know that leadership in rare disease is deeply personal. Our thoughts are with Doug and his family, especially as they navigate their own journey with myotonic dystrophy. We know how challenging this path can be, and we’re wishing them strength and support in the days ahead.

Every step forward matters, and we remain committed to working alongside leaders and the Duchenne community to keep driving research forward for everyone affected.

02/26/2026

Entrada Therapeutics shares quarterly newsletter with recent updates on their Duchenne exon-skipping programs
https://mailchi.mp/entradatx/entradas-quarterly-community-newsletter-february-2026

02/25/2026

FUTURES 2026 | May 21–24 | Orlando, Florida

There’s no party like a FUTURES party 🥳

This year we’re turning up the glow with a NEON theme—so bring the brightest vibes, your boldest colors, and get ready to light up the night. 💙 Let’s celebrate, connect, and make unforgettable memories together in Orlando! 🌟

Register today using the link below https://web.cvent.com/event/ee9e7402-2c59-420f-9cf9-5731cf20d4d9/regProcessStep1

02/25/2026

Honored to celebrate an extraordinary evening at the EURORDIS Black Pearl Awards ✨

Debra Miller, CEO of CureDuchenne, with Robert and Trude Steen — the inspiring parents of Mats Steen, whose remarkable life is beautifully captured in The Remarkable Life of Ibelin.

Today, the documentary took home the Media and Awareness Raising Award, shining a powerful light on legacy, love, and the impact one life can have on the world. 💙

Congratulations to the Steen family, director Benjamin Ree, and all who continue to elevate the voices of the rare disease community.

02/23/2026

Roche has decided to stop recruitment for their Phase 2 SHIELD DMD study of satralizumab in Duchenne, citing the decision was not due to any new efficacy or safety issues, but rather due to feasibility concerns with meeting regulatory requirements, as well as recruitment and study completion deadlines.

Please see the community letter for more information, including next steps for study participants and individuals who are currently screening to participate. Roche plans to continue the study through the 6-month bone density data collection, which is expected in H2 2026.

We recognize that it is difficult news for the community anytime a study is discontinued, and want to thank all of the families who have been participating in this study.

Read more:

Roche has decided to stop recruitment for their Phase 2 SHIELD DMD study

02/23/2026

Donate today at https://fundraise.givesmart.com/e/3qoxXw?vid=1oub6f 💙

02/22/2026

🤠 What an incredible weekend at our Dallas Workshop!!

A heartfelt thank you to Dr. Diana Castro and her amazing team for welcoming our families and sharing their compassionate clinic with the Duchenne community. We are so grateful for your partnership and dedication.

We also extend our sincere thanks to Dr. Tiffany Garcia for her insightful nutrition presentation, Dr. Michael Kelly for sharing updates on the evolving treatment landscape, and Doug Levine and Kayla Clem for their valuable PT talk and practical guidance for families. Thank you to Sheryl and Rosalia for courageously sharing their family journeys, and to Jordan Lynch and Kate Miner Moebel for inspiring families with meaningful ways to get involved with CureDuchenne and help spread awareness. We also appreciate NuMotion for bringing equipment and providing hands-on support for our community.

And we couldn’t do this important work without the generous support of our sponsors — thank you for helping make this workshop possible and for standing with the Duchenne community.

We are so thankful to everyone who joined us in Dallas. Together, we continue to learn, connect, and move closer to a cure.

02/20/2026

For families facing Duchenne, access to truly expert care can change everything 💙

We are proud to celebrate the expansion of the CureDuchenne Clinic and the Neurology and Neuromuscular Care Center in greater Dallas. Because when a child is diagnosed with Duchenne or Becker muscular dystrophy, the last thing a family should have to fight for is access to coordinated, compassionate care.

Under the leadership of the incredible Neurology and Neuromuscular Care Center - Diana Castro MD , this clinic is redefining what comprehensive neuromuscular care looks like — bringing neurology, cardiology, pulmonology, physical therapy, diagnostics, and more under one roof. The clinic’s growth reflects the need and our shared commitment to meeting it.

Thank you to Cheryl Moore, Mayor of Flower Mound, for recognizing the importance of specialized care for families in your city. Thank you to Dr. Diana Castro, Dr. Shai Manzuri, and the entire clinic team for your extraordinary leadership and dedication to this community. And thank you to our generous donors who make this kind of care possible.

This is how we move progress forward - not only accelerating treatments, but raising the standard of care for everyone living with Duchenne.

02/20/2026

In honor of National Caregivers Day, we want to give a shoutout to the incredible caregivers who show up every day with strength, compassion, and love. 💙

From family caregivers supporting loved ones, to professional caregivers—nurses, and healthcare teams, and the volunteers who step in to help friends and neighbors—this day is for you.

Caregiving is demanding and often isolating work. Today, we shine a light on your dedication, acknowledge your sacrifices, and remind you that you are seen, valued, and deeply appreciated.

Together we will cure Duchenne 💙