Bettencourt, Brian R.

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Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study

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An RNAi therapeutic targeting Tmprss6 decreases iron overload in Hfe−/− mice and ameliorates anemia and iron overload in murine β-thalassemia intermedia

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Preclinical evaluation of RNAi as a treatment for transthyretin-mediated amyloidosis

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