Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study

BACKGROUND: Transthyretin-mediated amyloidosis is an inherited, progressively debilitating disease caused by mutations in the transthyretin gene. This study evaluated the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple doses of patisiran (ALN-TTR02), a small interfering RNA...

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Detalles Bibliográficos
Publicado en:Orphanet J Rare Dis
Autores principales: Suhr, Ole B, Coelho, Teresa, Buades, Juan, Pouget, Jean, Conceicao, Isabel, Berk, John, Schmidt, Hartmut, Waddington-Cruz, Márcia, Campistol, Josep M., Bettencourt, Brian R., Vaishnaw, Akshay, Gollob, Jared, Adams, David
Formato: Artigo
Lenguaje:Inglês
Publicado: BioMed Central 2015
Materias:
Research
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC4559363/
https://ncbi.nlm.nih.gov/pubmed/26338094
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-015-0326-6
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