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).

The first year after our son’s Duchenne diagnosis was difficult, confusing, and frustrating. We didn’t know where to turn, what questions to ask, and how to advocate for our son in a real and meaningful way...

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

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.