When the boys were diagnosed with DMD we understood that we needed to find a new home...

Cure Rare Disease’s mission is to develop advanced therapeutics to treat individuals with rare and ultra-rare genetic diseases including Duchenne muscular dystrophy (Duchenne). 

We love aquatic therapy and we love Swim Angelfish, a swim therapy program in our area. Our 4 year old son, Ned, has been a devoted "swimmer" for nearly 2 years. Not only is swimming wonderful exercise and a meaningful confidence builder, the angelfish team is exceptional.

Unlike traditional methods for developing therapeutics for common and (relatively) rare diseases, ultra-rare disease drug development requires a different approach and mechanism in light of a limited ability to commercialize and even reach BLA/NDA approval once clinical trials are complete....