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Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis | Synapse

July 4, 2018New England Journal of Medicine3,001 citationsOpen Access

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Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

DADavid AdamsInterventional / Structural Cardiology AGAlejandra González‐DuarteNYU Langone Health WOWilliam O’RiordanUniversité Claude Bernard Lyon 1

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Resumen

In this trial, patisiran improved multiple clinical manifestations of hereditary transthyretin amyloidosis. (Funded by Alnylam Pharmaceuticals; APOLLO ClinicalTrials.gov number, NCT01960348 .).

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Cite This Study

Adams et al. (Wed,) studied this question.

synapsesocial.com/papers/69d6893b2c8f994bcf1f2eb3 https://doi.org/https://doi.org/10.1056/nejmoa1716153

Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

1Trial design and rationale for APOLLO, a Phase 3, placebo-controlled study of patisiran in patients with hereditary ATTR amyloidosis with polyneuropathy2017 · 179 citations
2Long-term treatment of transthyretin familial amyloid polyneuropathy with tafamidis: a clinical and neurophysiological study2016 · 108 citations
3Challenges and Opportunities for Local and Systemic Delivery of siRNA and Antisense Oligonucleotides2008 · 65 citations
4Preliminary assessment of neuropathy progression in patients with hereditary ATTR amyloidosis after orthotopic liver transplantation (OLT)2015 · 12 citations
5Tafamidis for transthyretin familial amyloid polyneuropathy2012 · 806 citations