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.

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....

Elijah Stacy is the founder of the 501(c)(3) nonprofit Destroy Duchenne, which he started at the age of fifteen. He is determined to advance gene editing and gene therapy to save his life, his brother’s life, and the lives of thousands of people around the globe who have been diagnosed with 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.

Gene therapy is the future of treating rare, genetic diseases that were previously thought to have no cure. CRD is breaking down what gene therapy is and how we are using gene therapy to develop therapies for neuromuscular diseases including Duchenne muscular dystrophy (DMD), limb-girdle muscular dystrophy subtypes, and spinocerebellar ataxia type 3 (SCA3).