One year ago, ITF's first treatment option became available in the U.S. for eligible individuals living with Duchenne muscular dystrophy. In the twelve months since, we’ve reached more than 1,000 families with our therapy – each with their own story, their own fight, their own hope. That number represents far more than a milestone. It’s an acknowledgment that we haven’t reached thousands more families who may benefit and a reflection that we have more work to do to realize our vision: a future where all families affected by rare disease have meaningful treatment options and the information they need to make sound decisions. It’s also a reminder of the dire need faced by families living with Duchenne and a call to action to keep moving forward urgently – with and for this community.

One thousand lives touched is not just a number. Thanks to the strong voices of this community, we are constantly reminded that behind each statistic is a person, a family, a future. Families, advocates, and clinicians help guide our way by sharing their experiences, expertise, and insights. The Duchenne community has partnered with us, supported us, and – most importantly – challenged us with meaningful feedback on how to improve, and we are deeply grateful for your collaboration.

One thousand lives touched is not an isolated measure of impact. While we focus on helping families make educated treatment decisions, we also show up as community partners in other ways. We’re honored to support programs like the Jett Foundation Giving Fund, which helps families secure accessible vehicles and the U.S. Volt Hockey Association, which expands opportunities for disabled athletes. We also sponsor critical work led by advocacy groups including Parent Project Muscular Dystrophy, CureDuchenne, Muscular Dystrophy Association, Team Joseph, Little Hercules and The Akari Foundation. These initiatives are not separate from our mission – they are core to it.

One thousand lives touched is a reminder of what’s possible when we listen, collaborate, and remain focused on why we are here. There is still so much more to do: more people to reach, more questions to answer, more science to push forward. Our work continues with urgency and purpose because people living with Duchenne don’t have time to wait.

Two eloquent community leaders, moms to Charlie and Jackson, recently issued a powerful call to action when they wrote:

Families, clinicians, policymakers, and industry should work collectively to apply lessons learned from new therapies – both tragedies and triumphs – to sustain and multiply progress for the sake of patients who lack the luxury of time.

For Kelly and Jennifer’s sons, and for every person with Duchenne, that call is heard loud and clear by the ITF team. We recognize this moment not as a finish line, but as a chance to reflect on progress and recommit ourselves to what comes next.

C-DUV-US-0537 08/2025