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...

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.

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

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.…

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....