Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS
Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS
About this item
Full title
Author / Creator
Miller, Timothy , Cudkowicz, Merit , Shaw, Pamela J , Andersen, Peter M , Atassi, Nazem , Bucelli, Robert C , Genge, Angela , Glass, Jonathan , Ladha, Shafeeq , Ludolph, Albert L , Maragakis, Nicholas J , McDermott, Christopher J , Pestronk, Alan , Ravits, John , Salachas, François , Trudell, Randall , Van Damme, Philip , Zinman, Lorne , Bennett, C. Frank , Lane, Roger , Sandrock, Alfred , Runz, Heiko , Graham, Danielle , Houshyar, Hani , McCampbell, Alexander , Nestorov, Ivan , Chang, Ih , McNeill, Manjit , Fanning, Laura , Fradette, Stephanie and Ferguson, Toby A
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
In a phase 1–2 dose-escalation trial involving adults with ALS due to
SOD1
mutations who received intrathecal tofersen (an antisense oligonucleotide) or placebo, the levels of mutant SOD1 in the CSF were 33 percentage points lower in the highest-dose tofersen group than in the placebo group.
Alternative Titles
Full title
Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS
Authors, Artists and Contributors
Author / Creator
Cudkowicz, Merit
Shaw, Pamela J
Andersen, Peter M
Atassi, Nazem
Bucelli, Robert C
Genge, Angela
Glass, Jonathan
Ladha, Shafeeq
Ludolph, Albert L
Maragakis, Nicholas J
McDermott, Christopher J
Pestronk, Alan
Ravits, John
Salachas, François
Trudell, Randall
Van Damme, Philip
Zinman, Lorne
Bennett, C. Frank
Lane, Roger
Sandrock, Alfred
Runz, Heiko
Graham, Danielle
Houshyar, Hani
McCampbell, Alexander
Nestorov, Ivan
Chang, Ih
McNeill, Manjit
Fanning, Laura
Fradette, Stephanie
Ferguson, Toby A
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_swepub_primary_oai_DiVA_org_umu_175822
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_swepub_primary_oai_DiVA_org_umu_175822
Other Identifiers
ISSN
0028-4793,1533-4406
E-ISSN
1533-4406
DOI
10.1056/NEJMoa2003715
