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Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS

by Genge, Angela , Shaw, Pamela J. , Van Damme, Philip , Ferguson, Toby A. , Ludolph, Albert C. , Chew, Sheena , Graham, Danielle , Chary, Sowmya , Bucelli, Robert C. , McDermott, Christopher J. , Andrews, Jinsy A. , McNeill, Manjit , Cudkowicz, Merit E. , Benatar, Michael , Wu, Fan , Sun, Peng , Chiò, Adriano , Zhu, Han , Miller, Timothy M. , Nestorov, Ivan , Glass, Jonathan D. , Cochrane, Thos , Babu, Suma , Fradette, Stephanie , Fanning, Laura , Sobue, Gen

in Adverse events / Amyotrophic lateral sclerosis / Antisense oligonucleotides / Antisense therapy / Cerebrospinal fluid / Drug therapy / Genetics / Genetics General / Light chains / Muscles / Mutation / Neurology / Neuromuscular Disease / Neurosurgery / Placebos / Proteins / Questionnaires / Superoxide dismutase / Ventilation

2022

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Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS

2022

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Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS

2022

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Journal Article

Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS

Genge, Angela,

Shaw, Pamela J.,

Van Damme, Philip,

Ferguson, Toby A.,

Ludolph, Albert C.,

Chew, Sheena,

Graham, Danielle,

Chary, Sowmya,

Bucelli, Robert C.,

McDermott, Christopher J.,

Andrews, Jinsy A.,

McNeill, Manjit,

Cudkowicz, Merit E.,

Benatar, Michael,

Wu, Fan,

Sun, Peng,

Chiò, Adriano,

Zhu, Han,

Miller, Timothy M.,

Nestorov, Ivan,

Glass, Jonathan D.,

Cochrane, Thos,

Babu, Suma,

Fradette, Stephanie,

Fanning, Laura,

Sobue, Gen

2022

Overview

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.

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Publisher

Massachusetts Medical Society

Subject

Adverse events

/ Amyotrophic lateral sclerosis

/ Antisense oligonucleotides

/ Antisense therapy

/ Cerebrospinal fluid

/ Drug therapy

/ Genetics