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). 

When the boys were diagnosed with DMD we understood that we needed to find a new home...

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…