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Twenty-one patients treated over 612 months showed benefit in biochemical markers

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Original research articles were included if they offered novel mechanistic insights or translational data on a qualifying compound

DOI 10.1016/S0140-6736(18)31773-2 - phase 2 daily-injection dose finding

The highest h--Gal A activity was observed in the liver, spleen, and plasma, where it increased more than 8-fold, while activity in the heart and kidneys increased 2- to 3-fold 24 h post-injection