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Requires consistent use over weeks for visible benefits

Spherical nucleic acid nanoparticle conjugates as an RNAi-based therapy for glioblastoma

FXS results from an expanded CGG repeat sequence, which may include over 200 repeats (so-called full mutation) in the 5 untranslated region of the FMR1 gene situated at Xq27.3

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This means its powerful effects are achieved not through mass saturation, but by rapidly activating intracellular pathways [3]

However, if the FDA ultimately reclassifies them and they are produced by compounding pharmacies in the U.S., clinicians would have discretion to write prescriptions outside of those narrow indications