Now, the wonderful Adam Feuerstein posts a perfect example of Acts of The Totally Unhinged
The Duchenne muscular dystrophy [DMD] patient & advocacy community keeps a gene therapy and multiple drugs on the US market, stopping the FDA from taking action, despite failed confirmatory clinical trials showing no efficacy and questionable safety.A bit of background. Under pressure from many patient/advocate communities to loosen up the approval process for rare and/or unmet needs, FDA did adopt some 'looser' regulations. DMD was among the recent cases where highly questionable drugs gained approval.
The Huntington's disease patient and advocacy community can't even persuade the FDA to allow a filing of a gene therapy, despite a positive clinical trial.
What Feuerstein is referring to is FDA's two-faced behavior: really stupid DMD drugs get approved on lousy data, but Huntington's developers get cut no slack even with actionable data. For myself, I smell the rancid odor of Baksheesh.
Thank you Ayatollah 49% Don© for your attention to this matter.

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