Hear community perspectives on topics ranging from navigating a diagnosis to reflecting on clinical trial experiences.
We’re all familiar with what the news is saying about COVID and its effect, but how does it really affect rare disease patients and their families? We asked Jess Curran and Karen Morales to share the experiences they had when COVID made its way to their families.
Cure Rare Disease’s mission is to develop advanced therapeutics to treat individuals with rare and ultra-rare genetic diseases including Duchenne muscular dystrophy (Duchenne).
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…