Hear community perspectives on topics ranging from navigating a diagnosis to reflecting on clinical trial experiences.
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.
We love aquatic therapy and we love Swim Angelfish, a swim therapy program in our area. Our 4 year old son, Ned, has been a devoted "swimmer" for nearly 2 years. Not only is swimming wonderful exercise and a meaningful confidence builder, the angelfish team is exceptional.
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…