Compositions for treating and/or preventing protein-aggregation diseases

Abstract

Proteopathies cover a wide spectrum of afflictions, including neurodegenerative diseases (e.g., Alzheimer's, Parkinson's, polyglutamine diseasessuch as Huntingtin in Huntington's disease, prion diseases); amyloidosis of other non-nervous system proteins such as Į1-antitrypsin, immunoglobulin light and heavy chains, lactadherin, apolipoprotein, gelsolin, lysozyme, fibrinogen, atrial natriuretic factor, keratin, lactoferrin and beta-2 microglobulin, among others); sickle cell disease; cataracts; cystic fibrosis; retinitis pigmentosa; and nephrogenic diabetes insipidus.Surprisingly, the administration of a sulfatase inhibitor is suitableto treat and/or prevent the proteotoxicity commonly associated with proteopathies. The present invention, therefore, provides compositions comprising sulfatase inhibitors for the treatment of proteopathies.