Regenxbio hits Duchenne gene therapy milestone, eyes 2027 FDA approval

Regenxbio has announced that its gene therapy for Duchenne muscular dystrophy has successfully met the requirements in a pivotal study, positioning the company to seek FDA approval by 2027. If successful, Regenxbio aims to become the second company to bring this treatment to market, potentially offering new hope for patients with this debilitating condition.