Excellent NYT article on reluctant FDA The F.D
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The F.D.A. can have a more flexible view of treatment efficacy without losing focus on safety . As with any drug, whether for migraines or asthma, there will be a spectrum of effectiveness. The same will be true of all gene therapies, and the F.D.A. should reconsider the metrics it uses to green-light these treatments now, before it potentially leaves thousands of patients in the lurch, out of access to something lifesaving.
https://www.nytimes.com/2024/02/19/opinion/ge...vidys.html