Transthyretin Antisense Oligonucleotide ISIS-TTR Rx
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Epidemiological, genetic association, and Mendelian randomization studies have provided strong evidence that lipoprotein (a) [Lp… Expand Abstract Transthyretin amyloidosis (ATTR amyloidosis) is a rare disease that results from the deposition of misfolded… Expand Characterization of a thermal neutron beam monitor based on gas electron multiplier technology Gabriele Croci1,4,∗, Carlo… Expand Opinion statementSpinal muscular atrophy is caused by mutations in the survival motor neuron 1 (SMN1) gene, leading to the… Expand This open-label, multiple ascending-dose study was conducted to evaluate the safety, tolerability, pharmacokinetics… Expand Objective: This study was conducted to evaluate the safety, tolerability, pharmacokinetics, and clinical efficacy of multiple… Expand This study was conducted to evaluate the safety, tolerability, pharmacokinetics, and clinical efficacy of multiple intrathecal… Expand Transthyretin (TTR)-associated amyloidosis is a late-onset autosomal-dominant genetic disease. Over 100 amyloidogenic mutations… Expand Summary form only given. Standard optical coherence tomography (OCT) has been proven to provide surface and general 3D metrology… Expand Five new suberosane sesquiterpenes, suberosenol A (1), suberosenol B (2), suberosanone (3), suberosenol A acetate (4), and… Expand