Hear community perspectives on topics ranging from navigating a diagnosis to reflecting on clinical trial experiences.
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 Sunday, October 29, 2023, our team joined the Tckachenko family and community of Scottsdale, Arizona for the inaugural community fundraiser organized by Heal DMD - a nonprofit organization committed to promoting healing and well-being for individuals with Duchenne Muscular Dystrophy (DMD) and their families....
Jessica Curran is a DMD mother to her son Conner, CRD board member as well as a DMD patient advocate.