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.

The first year after our son’s Duchenne diagnosis was difficult, confusing, and frustrating. We didn’t know where to turn, what questions to ask, and how to advocate for our son in a real and meaningful way...

Jessica Curran is a DMD mother to her son Conner, CRD board member as well as a DMD patient advocate.

An Interview With Seda Filenko As one of the first families dosed with gene therapy, what were some of your thoughts, feelings, hopes, fears, anything you really considered as you made the decision to enroll in the trial? The idea…