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Paper of the month: Improving neuropathy in hereditary transthyretin amyloidosis by targeting mRNA transcription
For October 2018, we have selected two papers. The first is: Adams D, Gonzales-Duarte A, O’Riordan WD, et al. Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis.(APOLLO trial). NEJM 2018;179:11-21. The second is: Benson MD, M Waddington-Cruz, JL Berk, et al. Inotersen treatment for patients with hereditary transthyretin amyloidosis. (NEURO-TTR trial) NEJM 2018;179:22-31.
Hereditary transthyretin amyloidosis is a rare autosomal dominant disorder in which mutations of the gene for transthyretin protein (TTR) induce systemic deposition of amyloid.