Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense Oligonucleotide AKCEA-TTR-L<sub>Rx</sub> (ION-682884-CS3) in Hereditary Transthyretin-Mediated Amyloid Polyneuropathy.

Neurol Ther · 2021 · PMC8140170 · PMID 33638113

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highly significantno p-value reported

In the NEURO-TTR study, the mNIS + 7 scoring approach showed highly significant improvement in global scores and essentially all sub-scores with inotersen treatment, including quantitative measures and potentially objective measures in scored neuropathy signs [ 26 , 35 ].

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