Methods and compositions for the treatment of amyloidosis
AssigneeUltragenyx Pharmaceutical Inc.
InventorsEmil D. KAKKIS, Michel Claude Vellard, Andrzej Swistowski
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Abstract
Methods and compositions for the treatment or prevention of amyloidosis are provided. In some embodiments, the methods comprise administering to the subject a therapeutically effective amount of at least one catabolic enzyme or a biologically active fragment thereof. Such methods and compositions may be employed to reduce, prevent, degrade and/or eliminate amyloid formation in the lysosome and/or extracellularly.
Claims20
A method of treating or preventing AL amyloidosis in a subject comprising administering to the subject a composition comprising a therapeutically effective amount of at least one catabolic enzyme or a biologically active fragment thereof.
A composition for treating or preventing AL amyloidosis in a subject, the composition comprising at least two catabolic enzymes, wherein the composition comprises at least one catabolic enzyme that is targeted to the cell lysosome and at least one catabolic enzyme that remains outside the cell.
Dependencies are inferred from the claim text.