TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS

The disclosure provides the use of neurofilament light chain levels for selecting a subject with a mutation in the superoxide dismutase 1 (SOD 1) gene for treatment with a SOD 1-targeting antisense oligonucleotide or salt thereof. The disclosed methods can be used in the treatment amyotrophic latera...

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Hauptverfasser: Fradette, Stephanie Melillo, Sun, Peng, Wong, Janice Chun Yee, Liu, Yingying, Graham, Danielle LeeZetta
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creator Fradette, Stephanie Melillo
Sun, Peng
Wong, Janice Chun Yee
Liu, Yingying
Graham, Danielle LeeZetta
description The disclosure provides the use of neurofilament light chain levels for selecting a subject with a mutation in the superoxide dismutase 1 (SOD 1) gene for treatment with a SOD 1-targeting antisense oligonucleotide or salt thereof. The disclosed methods can be used in the treatment amyotrophic lateral sclerosis, including clinically presymptomatic amyotrophic lateral sclerosis.
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subjects BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
SPIRITS
VINEGAR
WINE
title TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS
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