An evaluation of patisiran: a viable treatment option for transthyretin-related hereditary amyloidosis.

Milani, Paolo; Mussinelli, Roberta; Perlini, Stefano; Palladini, Giovanni; Obici, Laura

Milani, Paolo; Mussinelli, Roberta; Perlini, Stefano; Palladini, Giovanni; Obici, Laura.

Afiliação

Milani P; Amyloidosis Research and Treatment Center, Foundation IRCCS Policlinico San Matteo, Department of Molecular Medicine, University of Pavia, Pavia, Italy.
Mussinelli R; Amyloidosis Research and Treatment Center, Foundation IRCCS Policlinico San Matteo, Department of Molecular Medicine, University of Pavia, Pavia, Italy.
Perlini S; Amyloidosis Research and Treatment Center, Foundation IRCCS Policlinico San Matteo, Department of Molecular Medicine, University of Pavia, Pavia, Italy.
Palladini G; Emergency Department, Foundation IRCCS Policlinico San Matteo, Department of Internal Medicine, University of Pavia, Pavia, Italy.
Obici L; Amyloidosis Research and Treatment Center, Foundation IRCCS Policlinico San Matteo, Department of Molecular Medicine, University of Pavia, Pavia, Italy.

Expert Opin Pharmacother ; 20(18): 2223-2228, 2019 Dec.

Article em En | MEDLINE | ID: mdl-31566422

Assuntos

Neuropatias Amiloides Familiares/terapia; RNA Interferente Pequeno/uso terapêutico; Cardiomiopatias/terapia; Humanos; Mutação; Polineuropatias/terapia; Qualidade de Vida; Interferência de RNA

Palavras-chave

Patisiran; RNA interference; cardiomiopathy; hereditary transthyretin mediated amyloidosis; peripheral neuropathy

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Neuropatias Amiloides Familiares / RNA Interferente Pequeno Idioma: En Ano de publicação: 2019 Tipo de documento: Article

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