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.

CRISPR mediated exon-skipping is a method of genome editing that can be used to develop life-saving therapeutics for people with rare and ultra-rare genetic disorders. CRD is explaining the science behind these technologies as well as how they are the key to treating these diseases.