TY  - GEN
AU  - Suhr, Ole B
AU  - Coelho, Teresa
AU  - Bonilla, Alfonso
AU  - Pouget, Jean
AU  - Conceicao, Isabel
AU  - Berk, John
AU  - Schmidt, Hartmut
AU  - Waddington-Cruz, Marcia
AU  - Campistol, Josep M
AU  - Bettencourt, Brian R
AU  - Vaishnaw, Akshay
AU  - Gollob, Jared
AU  - Adams, David
PY  - 2015
DO  - 10.1186/s13023-015-0326-6
SN  - 1750-1172
UR  - http://hdl.handle.net/20.500.12105/20141
AB  - 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...
LA  - eng
PB  - BioMed Central (BMC)
KW  - Patisiran
KW  - RNA interference
KW  - Transthyretin-mediated familial amyloidotic polyneuropathy
KW  - Polyneuropathy
KW  - Hereditary disease
KW  - Genetic mutation
KW  - Phase II
KW  - Clinical trial
KW  - RNA, Small Interfering
KW  - Aged
KW  - Male
KW  - Dose-Response Relationship, Drug
KW  - Female
KW  - Humans
KW  - Middle Aged
KW  - Amyloid Neuropathies, Familial
TI  - Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study
TY  - research article
ER  -