US Patent 11786546 Compounds and methods for modulating GFAP
Patent 11786546 was granted and assigned to Ionis Pharmaceuticals, Inc. on October, 2023 by the United States Patent and Trademark Office.
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US Patent 11786546 Compounds and methods for modulating GFAP
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of GFAP RNA in a cell or subject, and in certain instances reducing the amount of GFAP in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a leukodystrophy. Such symptoms and hallmarks include motor delays, cognitive delays, paroxysmal deterioration, seizures, vomiting, swallowing difficulties, ataxic gait, palatal myoclonus, autonomic dysfunction, and presence of intra-astrocytic inclusions called Rosenthal fibers. Such leukodystrophies include Alexander Disease.
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