Our mission is to develop and fund life-saving genetic therapies for ultra-rare diseases, transforming the lives of patients previously deemed untreatable. By fostering strategic collaborations among top researchers, clinicians, policy experts, and donors, we bring hope and advanced solutions to those awaiting a cure.
In 1998, my brother, Terry, was diagnosed with Duchenne muscular dystrophy (DMD). He was three years old. We knew it was an unstoppable disease and we knew the clock was ticking for Terry.
The emerging science behind precision medicine and gene therapy sparked real hope that Terry might have a chance. However, the existing drug development infrastructure still wasn’t going to cut it for Terry. Unless we built something new.
In just three years, we developed a treatment for Terry’s rare mutation of DMD. Heartbreakingly, we were not able to stop the disease for Terry.
He passed away during the early stages of the clinical trial— an outcome that likely would have been avoided had the treatment been accessible sooner.
Our story does not end here. It can’t. Because there are hundreds of thousands of Terrys waiting for their chance to hit back. His legacy is a future where no family has to hear, “there’s nothing we can do.”
Cure Rare Disease's accomplishments are being recognized in the media
Your involvement helps shape our research and ensures our therapies meet real patient needs.
Cure Rare Disease, a non-profit organization (Tax ID number 82-2473513), is qualified as a tax-exempt organization under section 501(c)(3) of the IRS and has been designated as a “public charity” under section 170.
