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 (SOD1) gene for treatment with a SOD1-targeting antisense oligonucleotide or salt thereof. The disclosed methods can be used in the treatment amyotrophic lateral...

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Hauptverfasser: FRADETTE Stephanie Melillo, SUN Peng, GRAHAM Danielle Leezetta, LIU Yingying, WONG Janice Chun Yee
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creator FRADETTE Stephanie Melillo
SUN Peng
GRAHAM Danielle Leezetta
LIU Yingying
WONG Janice Chun Yee
description The disclosure provides the use of neurofilament light chain levels for selecting a subject with a mutation in the superoxide dismutase 1 (SOD1) gene for treatment with a SOD1-targeting antisense oligonucleotide or salt thereof. The disclosed methods can be used in the treatment amyotrophic lateral sclerosis, including clinically presymptomatic amyotrophic lateral sclerosis. La descripción proporciona el uso de niveles de cadenas ligeras de neurofilamentoss para seleccionar a un sujeto con una mutación en el gen de la superóxido dismutasa 1 (SOD1) para el tratamiento con un oligonucleótido antisentido dirigido a SOD1 o una sal de este. Los métodos descritos se pueden usar en el tratamiento de la esclerosis lateral amiotrófica, incluida la esclerosis lateral amiotrófica presintomática desde el punto de vista clínico.
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language eng ; spa
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subjects BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPIRITS
VINEGAR
WINE
title TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS
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