ITF’s first year of operations was unforgettable. We worked at breakneck speed to design and implement all the structures, processes, systems, and procedures necessary to bring a therapy to market. It has been gratifying to introduce a treatment option to a rare disease community that has been yearning, for far too long, for meaningful interventions. We have listened to and appreciated feedback from people affected by Duchenne muscular dystrophy (DMD) as we continue to focus on delivering value to the community. 

Heading into our second year of operations, two powerful family stories stick in my mind as signposts to guide us on our mission. The first is a video sent to us by the mother of a ten-year-old boy with DMD. The towheaded boy looks right at the camera and announces, “Here we go. Down the hatch!” He chugs the medicine, appears to pause for a second to consider the magnitude of the moment, then he breaks into a huge grin. When I watched that video for the first time, it all became real. It was emotional for all of us at ITF to see that video: We felt encouraged, we felt hopeful, we felt determined. We were doing some good. 

More recently, I received a phone call from a DMD mother whom I know. She was looking for more information than what we had available in our patient education materials. “You’re not showing up for this community in the way that you need to,” she admonished. “It’s not enough that you educate doctors; you are responsible to ensure that families have all the information we need to make decisions.” That was a sobering conversation. I hung up the phone with a strong take-home message: Good is not good enough. We need to reach higher and do better.   

With these stories front of mind, we have kicked off our second year with a clear vision for how to build upon our early accomplishments. Our first order of business is to better support the community we serve with more robust educational resources. We have just refreshed our educational materials for patients and physicians, and we are increasing our investment to make these resources more readily available to a broader audience.  

A second major focus for us this year is to generate clinical evidence that supplements the data from our pivotal trial. We are launching a new study to help us understand the real-world experience of individuals being treated with our medication. Simultaneously, we are working to further analyze the large volumes of information generated from our clinical trials. We value transparency and commit to sharing these data with the communities we serve.

We are 125% focused on doing right by the DMD community. At the same time, we are building an organization for the future, one that is equipped to serve other rare disease communities in need of better treatment options. We are investing in product development for other rare diseases, and we are engaging with various organizations looking for competent partners to bring new medicines to rare disease communities.

When I think back to just one year ago, I’m struck by how much can be accomplished in 12 months with the right people and a strong dose of passion. In January 2024, ITF was a team of two. Today, our team is strong and growing, with an approved therapy available in the US and UK. It has been a special privilege to be a part of this dedicated team. As I look ahead, I’m filled with hope and excitement for the impact we’re poised to make in 2025 and beyond.

C-DUV-US-0305 02/2025