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

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....

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....

​​We are proud to announce the creation of our Scientific Advisory Board (SAB)! Joining the SAB are leading experts in gene therapy and neuromuscular disorders who will help advance our preclinical and clinical development of gene therapy and antisense oligonucleotide programs.