CureDuchenne

04/10/2026

Today, we celebrate the incredible siblings in our CureDuchenne community 💙

You are advocates, teammates and best friends. Your role is powerful, and your impact is immeasurable.

We see you and we appreciate you.

Tag a sibling who deserves some extra love 👇💙

04/09/2026

FUTURES 2026 | May 21-24 | Orlando, Florida

In a article by MD news Griselle and Wilfredo, talk about their experience at FUTURES conference from a parent point of view.

“Our kids love the conferences,” Giselle says. “And we can meet other parents.”

But the connection doesn't end there, they mention how the families stay connected after and meet up every once in a while.

“We talk about clinical trials and about what’s going on in our lives. We have the oldest [child] in our group, so they ask us questions,” says Griselle. “It’s a good way we can share and listen.”

You can still join us a FUTURES in May. Register now using the link below!
https://web.cvent.com/event/ee9e7402-2c59-420f-9cf9-5731cf20d4d9/summary

04/08/2026

"It's time." After two decades of funding the science, advocating for her son, and refusing to give up, Debra Miller's words say it all. STAT News is telling the story of CureDuchenne, Hawken, and the next generation of exon-skipping therapies that are changing what's possible for Duchenne families.

An exon-skipping drug delivered striking results, but the Duchenne muscular dystrophy field has been burned before and some fear history could repeat. https://trib.al/WfPtwT4

04/07/2026

Our 2026 Impact report is out and worth a read!

Learn about Venture Philanthropy, the team, the impact and more using the link below 💙

https://cureduchenne.org/2026-impact-report/

04/06/2026

FUTURES 2026 | May 21-24 | Orlando, Florida

FUTURES is an annual conference by CureDuchenne to bring together the Duchenne community. It consists of sessions that will help families navigate this journey, by doctors, professionals, families and those living with Duchenne muscular dystrophy. But it’s not just a learning experience, there are also a lot of opportunities for families to connect with one another and for those who have Duchenne to find friends who truly understand them 💙

This years FUTURES conference is happening May 21-24 in Orlando, Florida.

It’s one that you don’t wanna miss! Check out the link below to learn more and register today ✨

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

04/05/2026

Wishing our CureDuchenne community a peaceful and hopeful Easter 💙🌷

This season reminds us of the strength, resilience, and progress that continues to move our mission forward—together.

We’re grateful for every family, advocate, and supporter who makes this community so powerful.

Here’s to brighter days ahead 💙

04/02/2026

FUTURES 2026 | May 21-24 | Orlando, Florida

FUTURES isn’t just a conference… it’s the place where our community shows up, connects, laughs, learns, and lifts each other up 🙌

From meeting other families,
To hearing the latest in research and care,
To unforgettable memories made together 💙

Spots are limited so register today using the link below!

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

Together we will cure Duchenne 💫

04/01/2026

PTC Therapeutics provided an update to the Duchenne community with additional details following the withdrawal of the ataluren New Drug Application (NDA).

The update notes that a limited remaining supply of Ataluren is still available, anticipated to last up to six months for individuals currently receiving treatment, and provides details for treating physicians to continue to get access to the drug. Following interactions with the FDA, PTC confirmed in February 2026 that it formally withdrew its NDA resubmission for ataluren, which the FDA has accepted. Ataluren is no longer under active review in the United States.

We know this news impacts families in our community. CureDuchenne remains committed to advancing research and supporting every individual and family navigating Duchenne.

Read the Community Letter:

PTC Therapeutics provided an update to the Duchenne community with additional details following the withdrawal of the ataluren New Drug Application (NDA).

03/30/2026

Our 2026 Impact report is out and worth a read!

Learn about our Mission, Impact, Community and more using the link below 💙

https://cureduchenne.org/event/2026-futures-national-conference/

03/29/2026

We’re still holding onto the feeling from our Baltimore workshop—what a truly special and meaningful day 💙

Hearing Jake Marrazzo share his Duchenne and life journey was incredibly moving and set the tone for a day rooted in honesty, strength, and connection. We’re so grateful to Dr. Andreas Barth from Johns Hopkins for thoughtfully walking us through cardiac considerations, and to Dr. Doris Leung from Kennedy Krieger for helping families better understand acute care management. Ann McCormick brought invaluable insight into physical therapy, while Numotion made it real by bringing equipment and hands-on support. And thank you to Lianna Orlando for guiding everyone through the Duchenne treatment landscape with clarity and care.

Most of all, we are deeply thankful to the families who chose to spend their Saturday with us—your openness, resilience, and willingness to connect are what make this community so powerful.

And to our sponsors, thank you for helping make a day like this possible.

We left Baltimore feeling inspired, connected, and reminded of why this work matters so much! 💙

03/27/2026

Thank you to Davia Jones for sharing your family's story with Mindy Basara WBAL and WBAL-TV 11 Baltimore!

Through CureDuchenne, Davia and Kyrie found a community that understands this journey and reminds families they are not alone.

Join Davia and our CureDuchenne Cares team at our workshop in Baltimore tomorrow as we bring the community together and continue driving progress forward for families.

Watch now: https://www.wbaltv.com/article/health-nonprofit-aids-son-duchenne-muscular-dystrophy/70869992

03/26/2026

To everyone who bid, raised their paddle, and showed up for the Duchenne community at Napa in Newport — thank you. And to the families who shared their stories: your courage is the reason we do this. 💙