Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS | Zendy

Jonathan Mill | Zendy; Merit Cudkowicz | Zendy; Pamela J. Shaw | Zendy; Peter M. Andersen | Zendy; Nazem Atassi | Zendy; Robert C. Bucelli | Zendy; Angela Genge | Zendy; Jonathan D. Glass | Zendy; Shafeeq Ladha | Zendy; Albert Ludolph | Zendy; Nicholas J. Maragakis | Zendy; Christopher McDermott | Zendy; Alan Pestronk | Zendy; John Ravits | Zendy; François Salachas | Zendy; Randall Trudell | Zendy; Philip Van Damme | Zendy; Lorne Zinman | Zendy; C. Frank Bennett | Zendy; Roger Lane | Zendy; Alfred Sandrock | Zendy; Heiko Runz | Zendy; Danielle Graham | Zendy; Hani Houshyar | Zendy; Alexander McCampbell | Zendy; Ivan Nestorov | Zendy; Ih Chang | Zendy; Manjit McNeill | Zendy; Laura Fanning | Zendy; Stephanie Fradette | Zendy; Toby A. Ferguson | Zendy

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Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS

Author(s) -

Jonathan Mill,

Merit Cudkowicz,

Pamela J. Shaw,

Peter M. Andersen,

Nazem Atassi,

Robert C. Bucelli,

Angela Genge,

Jonathan D. Glass,

Shafeeq Ladha,

Albert Ludolph,

Nicholas J. Maragakis,

Christopher McDermott,

Alan Pestronk,

John Ravits,

François Salachas,

Randall Trudell,

Philip Van Damme,

Lorne Zinman,

C. Frank Bennett,

Roger Lane,

Alfred Sandrock,

Heiko Runz,

Danielle Graham,

Hani Houshyar,

Alexander McCampbell,

Ivan Nestorov,

Ih Chang,

Manjit McNeill,

Laura Fanning,

Stephanie Fradette,

Toby A. Ferguson

Publication year - 2020

Publication title -

new england journal of medicine

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 19.889

H-Index - 1030

eISSN - 1533-4406

pISSN - 0028-4793

DOI - 10.1056/nejmoa2003715

Subject(s) - medicine , placebo , sod1 , amyotrophic lateral sclerosis , adverse effect , randomization , confidence interval , cerebrospinal fluid , pharmacokinetics , clinical endpoint , hazard ratio , anesthesia , gastroenterology , clinical trial , pathology , alternative medicine , disease

Tofersen is an antisense oligonucleotide that mediates the degradation of superoxide dismutase 1 (SOD1) messenger RNA to reduce SOD1 protein synthesis. Intrathecal administration of tofersen is being studied for the treatment of amyotrophic lateral sclerosis (ALS) due to SOD1 mutations.

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