abstract |
Proteopathy is a neurodegenerative disease (eg, polyglutamine disease such as huntingtin in Alzheimer's disease, Parkinson's disease, Huntington's disease, prion's disease); other non-neurological proteins (especially I1-antitrypsin, immunoglobulin light chain and heavy). Amyloidosis of chains, lactodoherin, apolipoprotein, gelzoline, lysozyme, fibrinogen, atrial sodium diuretic factor, keratin, lactoferrin and β-2 microglobulin); sickle red disease; cataracts; cystic fibrosis; retinal pigment degeneration; It also includes a wide range of distress, including renal proteopathy. Surprisingly, administration of sulfatase inhibitors is generally suitable for treating and / or preventing protein toxicity associated with proteopathy. Accordingly, the present invention provides compositions comprising sulfatase inhibitors for the treatment of proteopathy. [Selection diagram] None |