Hear community perspectives on topics ranging from navigating a diagnosis to reflecting on clinical trial experiences.
Unlike traditional methods for developing therapeutics for common and (relatively) rare diseases, ultra-rare disease drug development requires a different approach and mechanism in light of a limited ability to commercialize and even reach BLA/NDA approval once clinical trials are complete....
I’m Vivek Gohil, I’m 30, and I live with Duchenne Muscular Dystrophy. My life goal is to leave a mark, make the most of life, and help others along the way. Although it’s daunting, I never worry about the future.…
This past week, Cure Rare Disease’s first therapeutic 一developed using CRISPR technology to treat Duchenne muscular dystrophy一began its final stage of testing leading up to dosing this fall. This CRISPR therapeutic will be one of the first of its kind to be delivered systemically to the human body, an accomplishment built on decades of science and research. As we approach this huge milestone in CRD’s mission, we wanted to reflect on the path that has gotten us here today.