TY  - GEN
AU  - Coelho, Teresa
AU  - Adams, David
AU  - Conceicao, Isabel
AU  - Waddington-Cruz, Marcia
AU  - Schmidt, Hartmut H
AU  - Bonilla, Alfonso
AU  - Campistol, Josep
AU  - Berk, John L
AU  - Polydefkis, Michael
AU  - Wang, Jing Jing
AU  - Chen, Jihong
AU  - Sweetser, Marianne T
AU  - Gollob, Jared
AU  - Suhr, Ole B
PY  - 2020
DO  - 10.1186/s13023-020-01399-4
UR  - https://hdl.handle.net/20.500.12105/22860
AB  - Background: Patisiran, an RNA interference therapeutic, has demonstrated robust reduction of wild-type and mutant transthyretin protein and was able to improve polyneuropathy and quality of life following 18 months of treatment in patients with...
LA  - eng
PB  - BioMed Central (BMC)
KW  - ATTR amyloidosis
KW  - Cardiomyopathy
KW  - Patisiran
KW  - Polyneuropathy
KW  - RNA interference
TI  - A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis
TY  - research article
ER  -