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: LIU, Yingying, WONG, Janice Chun Yee, FRADETTE, Stephanie Melillo, GRAHAM, Danielle LeeZetta, SUN, Peng
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Sprache:eng ; fre
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creator LIU, Yingying
WONG, Janice Chun Yee
FRADETTE, Stephanie Melillo
GRAHAM, Danielle LeeZetta
SUN, Peng
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 présente invention concerne l'utilisation des niveaux de chaîne légère de neurofilament pour sélectionner un sujet présentant une mutation dans le gène de la superoxyde dismutase 1 (SOD1) pour un traitement avec un oligonucléotide antisens ciblant SOD1 ou un de ses sels. Les procédés de l'invention peuvent être utilisés dans le traitement de la sclérose latérale amyotrophique, y compris la sclérose latérale amyotrophique cliniquement présymptomatique.
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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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