I have been studying neuroscience and working in the field of neuromuscular disease for more than 20 years. I may have the academic degrees and the work experience to qualify as an "expert," but there's no doubt in my mind that the real experts are you – the people who live with their diagnosis every single minute of every single day.

Without the critical knowledge you bring to the table to complement my education and experience, my official expertise only goes so far. You wake up in the morning and feel the effects of DMD the moment you open your eyes and continue to feel them until you lay your head to rest at night – if rest is even a luxury you are afforded. Only you know how it feels to manage the day to day with Duchenne, and only you know what’s truly important to you and your family when making decisions about treatments, clinical trial participation, and interventions like stretching, aquatherapy, and nutritional supplements. Only you have firsthand experience of how life with DMD affects social relationships, education choices, the well-being of siblings, and other real-life experiences that are not fully captured in the medical literature.

That's why I need your cooperation and collaboration to do the best job I can here at ITF Therapeutics. As the Vice President, Head of US Medical Affairs, my mandate is to make sure physicians, patients, and caregivers have the tools they need to make well-informed treatment decisions. We contextualize information so that when healthcare providers and patients are trying to decide if a certain medicine is appropriate, they are well equipped to make a sound decision.

A few weeks ago I tore my Achilles tendon, and since I can’t walk I’ve been using a scooter to get around. For the first time in my life, I’ve been stressed about whether I’ll have enough stamina to make it through my day. I’m preoccupied with worry that I’ll encounter stairs or obstacles that will be impossible for me to navigate. The experience has been humbling. My injury hasn’t made me feel like now I understand what you must be going through. To the contrary, it underscores how little I know about what it’s like to live with a disease like DMD. It reminds me that countless challenges confront people living with DMD each and every day, and only you know what it feels like to manage them. And it reminds me of how important you are to this endeavor of bringing about new treatments that will result in healthier, happier lives.

So please, come to conferences, talk to us, share your stories, make your voices heard. To the dad who recently approached me at a conference to share his son’s clinical trial experience, thank you. The more input, insights, and questions we have from you to fuel our work, the better job we will do. So please share this post to spread the word: We need YOU!

C-DUV-US-0048 09/2024