Hear community perspectives on topics ranging from navigating a diagnosis to reflecting on clinical trial experiences.
My husband Keven and I have 3 kids and we’ve been homeschooling for more than 10 years. Here’s a little background of how we got here.
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).
CRISPR mediated exon-skipping is a method of genome editing that can be used to develop life-saving therapeutics for people with rare and ultra-rare genetic disorders. CRD is explaining the science behind these technologies as well as how they are the key to treating these diseases.