Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
About this item
Full title
Author / Creator
Adams, David , Gonzalez-Duarte, Alejandra , O’Riordan, William D , Yang, Chih-Chao , Ueda, Mitsuharu , Kristen, Arnt V , Tournev, Ivailo , Schmidt, Hartmut H , Coelho, Teresa , Berk, John L , Lin, Kon-Ping , Vita, Giuseppe , Attarian, Shahram , Planté-Bordeneuve, Violaine , Mezei, Michelle M , Campistol, Josep M , Buades, Juan , Brannagan, Thomas H , Kim, Byoung J , Oh, Jeeyoung , Parman, Yesim , Sekijima, Yoshiki , Hawkins, Philip N , Solomon, Scott D , Polydefkis, Michael , Dyck, Peter J , Gandhi, Pritesh J , Goyal, Sunita , Chen, Jihong , Strahs, Andrew L , Nochur, Saraswathy V , Sweetser, Marianne T , Garg, Pushkal P , Vaishnaw, Akshay K , Gollob, Jared A and Suhr, Ole B
Publisher
United States: Massachusetts Medical Society
Journal title
Language
English
Formats
Publication information
Publisher
United States: Massachusetts Medical Society
Subjects
More information
Scope and Contents
Contents
Hereditary transthyretin amyloidosis is caused by the deposition of misfolded transthyretin proteins in peripheral nerves and other tissues. This phase 3 trial tested patisiran, a small interfering RNA targeting transthyretin messenger RNA, to treat the disease.
Alternative Titles
Full title
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
Authors, Artists and Contributors
Author / Creator
Gonzalez-Duarte, Alejandra
O’Riordan, William D
Yang, Chih-Chao
Ueda, Mitsuharu
Kristen, Arnt V
Tournev, Ivailo
Schmidt, Hartmut H
Coelho, Teresa
Berk, John L
Lin, Kon-Ping
Vita, Giuseppe
Attarian, Shahram
Planté-Bordeneuve, Violaine
Mezei, Michelle M
Campistol, Josep M
Buades, Juan
Brannagan, Thomas H
Kim, Byoung J
Oh, Jeeyoung
Parman, Yesim
Sekijima, Yoshiki
Hawkins, Philip N
Solomon, Scott D
Polydefkis, Michael
Dyck, Peter J
Gandhi, Pritesh J
Goyal, Sunita
Chen, Jihong
Strahs, Andrew L
Nochur, Saraswathy V
Sweetser, Marianne T
Garg, Pushkal P
Vaishnaw, Akshay K
Gollob, Jared A
Suhr, Ole B
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_swepub_primary_oai_DiVA_org_umu_150728
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_swepub_primary_oai_DiVA_org_umu_150728
Other Identifiers
ISSN
0028-4793,1533-4406
E-ISSN
1533-4406
DOI
10.1056/NEJMoa1716153
