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Aubin Michalon et al.
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Jonathan C. Fox et al.
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Orly Moshe-Lilie et al.
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Masatoshi Minamisawa et al.
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Aaron Yarlas et al.
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David Adams et al.
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P. James B. Dyck et al.
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M. D. Benson et al.
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Aaron D. Springer et al.
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Andrea Cortese et al.
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Efficiency of silencing RNA for removal of transthyretin V30M in a TTR leptomeningeal animal model
Paula Goncalves et al.
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Suppressing transthyretin production in mice, monkeys and humans using 2nd-Generation antisense oligonucleotides
Elizabeth J. Ackermann et al.
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Nonbiopsy Diagnosis of Cardiac Transthyretin Amyloidosis
Julian D. Gillmore et al.
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Afro-Caribbean Heart Failure in the United Kingdom: Cause, Outcomes, and ATTR V122I Cardiac Amyloidosis
Jason N. Dungu et al.
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Thazha P. Prakash et al.
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