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: LIU, Yingying, WONG, Janice Chun Yee, FRADETTE, Stephanie Melillo, GRAHAM, Danielle LeeZetta, SUN, Peng
Format: Patent
Sprache:eng
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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.