Hear community perspectives on topics ranging from navigating a diagnosis to reflecting on clinical trial experiences.
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…
Gene therapy is the future of treating rare, genetic diseases that were previously thought to have no cure. CRD is breaking down what gene therapy is and how we are using gene therapy to develop therapies for neuromuscular diseases including Duchenne muscular dystrophy (DMD), limb-girdle muscular dystrophy subtypes, and spinocerebellar ataxia type 3 (SCA3).
When the boys were diagnosed with DMD we understood that we needed to find a new home...
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.…