‘It’s a vote for hope’: first gene therapy for muscular dystrophy nears approval, but will it work?

Another gene therapy for a deadly genetic neuromuscular disease, this time Duchenne muscular dystrophy, is nearing potential fda approval with a final decision expected this week. The scientific advisory panel narrowly voted in favor 8-6, but like in many fields currently the tension between early approvals on the basis of biomarkers and how that might effect the ability of researchers to obtain definitive measures of clinical efficacy in phase 3 trials remains.

New trial results have been seen at conferences and likely will be published publicly as well soon, previous small trial published here: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7296461/