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.

We’re all familiar with what the news is saying about COVID and its effect, but how does it really affect rare disease patients and their families? We asked Jess Curran and Karen Morales to share the experiences they had when COVID made its way to their families.

On Sunday, October 29, 2023, our team joined the Tckachenko family and community of Scottsdale, Arizona for the inaugural community fundraiser organized by Heal DMD - a nonprofit organization committed to promoting healing and well-being for individuals with Duchenne Muscular Dystrophy (DMD) and their families....

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