So Phase 3 has started! Would not indications of improvement in these girls be visible to Acadia and Neuren who are conducting the tests almost straight away? Would they not make decisions based on what they can see rather than wait another another one or two years for a regulator/bureaucracy (the FDA) to finally give the stamp of approval.
aurel19672003 in his/her post 40511561 described social media feedback from relatives on an earlier trial of NNZ-2566 (trofinitide). This trial was six long years ago! Wonder what those poor girls are doing now. Waiting and waiting for bureaucracy. I have been a holder of NEU over six years and have never seen a whisper of harmfull side effects from Trofinitide. Donald Trump has passed through "Right to Try" legislation to help people access medicines that they were unable to do previously.
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- Ann: Phase 3 trial of trofinetide in Rett syndrome commences
Ann: Phase 3 trial of trofinetide in Rett syndrome commences, page-44
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