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Trial design and rationale for APOLLO, a Phase 3, placebo-controlled study of patisiran in patients with hereditary ATTR amyloidosis with polyneuropathy

BACKGROUND: Patisiran is an investigational RNA interference (RNAi) therapeutic in development for the treatment of hereditary ATTR (hATTR) amyloidosis, a progressive disease associated with significant disability, morbidity, and mortality. METHODS: Here we describe the rationale and design of the P...

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Detalles Bibliográficos
Autores principales:	Adams, David, Suhr, Ole B., Dyck, Peter J., Litchy, William J., Leahy, Raina G., Chen, Jihong, Gollob, Jared, Coelho, Teresa
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2017
Materias:	Study Protocol
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5594468/ https://www.ncbi.nlm.nih.gov/pubmed/28893208 http://dx.doi.org/10.1186/s12883-017-0948-5

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