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...

​​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.