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.
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.
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....
Jessica Curran is a DMD mother to her son Conner, CRD board member as well as a DMD patient advocate.