The FDA’s decision, expected this month, follows several setbacks and delays and will pose difficult choices for the families of children with Duchenne muscular dystrophy.

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/

  • Daryl76679
    1·
    1 year ago

    Definitely interesting. I agree with the person who doesn’t envy the FDA right about now. Balancing hope and a potential cure or at least something that ameliorates the issue with caution is more than difficult. Doing that with everyone’s eyes on you especially so