Unlike traditional methods for developing therapeutics for common and (relatively) rare diseases, ultra-rare disease drug development requires a different approach and mechanism in light of a limited ability to commercialize and even reach BLA/NDA approval once clinical trials are complete....

My name is Katie Kuhl and I am a junior in high school right now. I am mostly focused on school and am involved in the math team, DECA, pre-med club, student council, and volunteering at my local animal shelter…

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…

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).