Hear community perspectives on topics ranging from navigating a diagnosis to reflecting on clinical trial experiences.
My name is Katie Kuhl and I am a junior in high school right now. I am mostly focused on school and am involved in the math team, DECA, pre-med club, student council, and volunteering at my local animal shelter…
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....
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.