Analysis of autonomic outcomes in APOLLO, a phase III trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis

Hereditary transthyretin-mediated (hATTR) amyloidosis is a progressive, debilitating disease often resulting in early-onset, life-impacting autonomic dysfunction. The effect of the RNAi therapeutic, patisiran, on autonomic neuropathy manifestations in patients with hATTR amyloidosis with polyneuropa...

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Publicat a:J Neurol
Autors principals: González-Duarte, Alejandra, Berk, John L., Quan, Dianna, Mauermann, Michelle L., Schmidt, Hartmut H., Polydefkis, Michael, Waddington-Cruz, Márcia, Ueda, Mitsuharu, Conceição, Isabel M., Kristen, Arnt V., Coelho, Teresa, Cauquil, Cécile A., Tard, Céline, Merkel, Madeline, Aldinc, Emre, Chen, Jihong, Sweetser, Marianne T., Wang, Jing Jing, Adams, David
Format: Artigo
Idioma:Inglês
Publicat: Springer Berlin Heidelberg 2019
Matèries:
Original Communication
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC7035216/
https://ncbi.nlm.nih.gov/pubmed/31728713
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s00415-019-09602-8
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