Hear community perspectives on topics ranging from navigating a diagnosis to reflecting on clinical trial experiences.
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.
I’m Vivek Gohil, I’m 30, and I live with Duchenne Muscular Dystrophy. My life goal is to leave a mark, make the most of life, and help others along the way. Although it’s daunting, I never worry about the future.…
On October 7th, the Baweja and Warner families hosted our inaugural Waves of Hope Wine Tasting event at the Ocean Institute in Dana Point, CA, raising more than $83,100 in support of CRD’s mission....
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....
Cure Rare Disease, in partnership with Charles River Laboratories, has successfully created 3 mouse models that harbor the humanized form of the dystrophin gene. Each model has a mutation that causes Duchenne muscular dystrophy that CRD is currently working to treat, meaning they can be utilized for critical in vivo studies to test the safety and efficacy of mutation-specific gene editing therapeutics. The success of this project paves the way for future therapeutics to be developed to treat rare and ultra-rare genetic disorders.