Last Thursday, the FDA voted to approve Sarepta's microdystrophin gene therapy for #Duchenne muscular #dystrophy.
https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-certain-patients-duchenne-muscular-dystrophy

This was not an easy call, made on the basis of a biomarker rather than efficacy data. I wrote about it last month for Nature Medicine.

#genetherapy #drugdiscovery #drugdevelopment

https://www.nature.com/articles/d41591-023-00052-4