Defining SOD1 ALS natural history to guide therapeutic clinical trial design.

doi:10.1136/jnnp-2016-313521

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Article

Defining SOD1 ALS natural history to guide therapeutic clinical trial design.

Journal of neurology, neurosurgery, and psychiatry - 1 Feb 2017

Bali Taha, Self Wade, Liu Jingxia, Siddique Teepu, Wang Leo H, Bird Thomas D, Ratti Elena, Atassi Nazem, Boylan Kevin B, Glass Jonathan D, Maragakis Nicholas J, Caress James B, McCluskey Leo F, Appel Stanley H, Wymer James P, Gibson Summer, Zinman Lorne, Mozaffar Tahseen, Callaghan Brian, McVey April L, Jockel-Balsarotti Jennifer, Allred Peggy, Fisher Elena R, Lopate Glenn, Pestronk Alan, Cudkowicz Merit E, Miller Timothy M

Abstract excerpt

IMPORTANCE: Understanding the natural history of familial amyotrophic lateral sclerosis (ALS) caused by SOD1 mutations (ALSSOD1) will provide key information for optimising clinical trials in this patient population. OBJECTIVE: To establish an updated natural history of ALSSOD1. DESIGN, SETTING AND PARTICIPANTS: Retrospective cohort study from 15 medical centres in North America evaluated records from 175...

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Identifiers

PMID: 27261500
DOI: 10.1136/jnnp-2016-313521
PMCID: PMC5136332

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SOD1

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

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