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

​​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.

On October 7th, the Baweja and Warner families hosted our inaugural Waves of Hope Wine Tasting event at the Ocean Institute in Dana Point, CA, raising more than $83,100 in support of CRD’s mission....

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....