Hear community perspectives on topics ranging from navigating a diagnosis to reflecting on clinical trial experiences.
We previously lived in Central California and raised organically fed pork on pasture. When Kal was diagnosed with Duchenne...
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.