Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS
Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS
About this item
Full title
Author / Creator
Miller, Timothy M. , Cudkowicz, Merit E. , Genge, Angela , Shaw, Pamela J. , Sobue, Gen , Bucelli, Robert C. , Chiò, Adriano , Van Damme, Philip , Ludolph, Albert C. , Glass, Jonathan D. , Andrews, Jinsy A. , Babu, Suma , Benatar, Michael , McDermott, Christopher J. , Cochrane, Thos , Chary, Sowmya , Chew, Sheena , Zhu, Han , Wu, Fan , Nestorov, Ivan , Graham, Danielle , Sun, Peng , McNeill, Manjit , Fanning, Laura , Ferguson, Toby A. and Fradette, Stephanie
Publisher
Boston: Massachusetts Medical Society
Journal title
Language
English
Formats
Publication information
Publisher
Boston: Massachusetts Medical Society
Subjects
More information
Scope and Contents
Contents
The antisense molecule tofersen was tested intrathecally in 108 patients with ALS due to
SOD1
mutations. In a subgroup with faster-progressing disease, there was no clinical difference from placebo over a period of 28 weeks.
Alternative Titles
Full title
Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS
Authors, Artists and Contributors
Author / Creator
Cudkowicz, Merit E.
Genge, Angela
Shaw, Pamela J.
Sobue, Gen
Bucelli, Robert C.
Chiò, Adriano
Van Damme, Philip
Ludolph, Albert C.
Glass, Jonathan D.
Andrews, Jinsy A.
Babu, Suma
Benatar, Michael
McDermott, Christopher J.
Cochrane, Thos
Chary, Sowmya
Chew, Sheena
Zhu, Han
Wu, Fan
Nestorov, Ivan
Graham, Danielle
Sun, Peng
McNeill, Manjit
Fanning, Laura
Ferguson, Toby A.
Fradette, Stephanie
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_proquest_miscellaneous_2716932393
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_2716932393
Other Identifiers
ISSN
0028-4793
E-ISSN
1533-4406
DOI
10.1056/NEJMoa2204705
