A devastating diagnosis changed everything for our family and fueled our mission to cure Duchenne muscular dystrophy, a fatal disease affecting 15,000 in the United States.
I’ll never forget the day my husband and I learned that our only child, Hawken, had Duchenne muscular dystrophy (DMD). The diagnosis came with a devastating prognosis: a degenerative neuromuscular disease with no cure. By age 12 he would lose the ability to walk, then lose use of his arms, and eventually face severe heart and lung complications. The doctor predicted he would not live past age 18.
Like most parents, we had never heard of Duchenne. Imagine receiving a diagnosis that chips away at your child’s future. It was one of over 10,000 rare diseases, yet 100% fatal. Our world and our purpose changed in that instant.
Initially, our desperation to find a cure for Hawken fueled our mission. My husband, Paul, and I created a nonprofit called CureDuchenne, determined to fund research and move scientific discoveries from the lab to clinical trials.
Along the way, we met countless families facing the same fears and heartbreak. We’ve been inspired by parents who are determined to provide the best possible care for their children and who refuse to give up despite their circumstances. Their strength transformed our perspective, and we knew we had a responsibility to fight for every child and family affected by this disease.
Achieving milestones and moving forward
Over the two decades since we created CureDuchenne, we have seen remarkable milestones, including the first FDA-approved exon-skipping drugs for Duchenne and a groundbreaking gene therapy approval. CureDuchenne has enabled much of this progress, investing in many research projects and helping bring 18 potential therapies to clinical trials. We’ve built the most experienced science team in the field, rigorously evaluating and investing in promising therapies that could transform lives.
These successes are significant, but they’re just the beginning.
Duchenne is a complex disease, and most individuals with Duchenne, including older individuals, still don’t have treatments that do enough to delay the progression of the disease. Duchenne’s variability highlights the need for personalized medicine, tailoring therapies to individual mutations and stages of progression.
We must continue to rally biotech and pharmaceutical companies to prioritize rare diseases like Duchenne. Developing treatments requires immense funding, and companies often deprioritize rare diseases due to limited disease knowledge and uncertain regulatory involvement.
Recently, I sat beside Hawken at a conference where much of the focus was on bringing treatments to younger boys. While progress for younger patients is vital, my heart breaks for older guys, like Hawken, to be overlooked after they’ve contributed so much to advance research. Their contributions must not — and will not — be forgotten.
Defying the odds
I’m proud to say that Hawken is now 28 years old with a thriving career after graduating from USC, and he serves as a constant reminder of what individuals can achieve even with the odds stacked against them. We’re here to ensure that individuals with Duchenne can do more than just survive — they can thrive.
As a mother, I will never stop fighting — for Hawken and for the thousands of families I’ve met along this journey. Families facing Duchenne and other rare diseases cannot fight this battle alone. It takes a global community — scientists, advocates, donors, and families — working together to turn hope into reality through funding, advocacy, and raising awareness. Together, we will not just imagine a cure — we will make it a reality.
