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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Bibliographische Detailangaben
Hauptverfasser:	FRADETTE Stephanie Melillo, SUN Peng, GRAHAM Danielle Leezetta, LIU Yingying, WONG Janice Chun Yee
Format:	Patent
Sprache:	eng ; spa
Schlagworte:	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
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Beschreibung
Zusammenfassung:	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.