Compositions and methods for the treatment of familial amyloid polyneuropathy

摘要

The invention relates to the compounds of formula I or its pharmaceutical acceptable salts, as well as polymorphs, solvates, enantiomers, stereoisomers and hydrates thereof. The pharmaceutical compositions comprising an effective amount of compounds of formula I, and methods for the treatment of familial amyloid polyneuropathy may be formulated for oral, buccal, rectal, topical, transdermal, transmucosal, intravenous, parenteral administration, syrup, or injection. Such compositions may be used to treatment of transthyretin-related hereditary amyloidosis, neurodegenerative diseases, amyloidosis, neuropathic pain, amyloid fibril formation and cardiomyopathy related diseases.

主权项

1. A compound of Formula I: or a pharmaceutically acceptable salt, hydrate, solvate, prodrug, enantiomer, or stereoisomer thereof, wherein: R1 represents H, D, CD3, OCH3, OCD3, R2 represents null, a is 2, 3 or 7; each b is independently 3, 5 or 6; e is 1, 2 or 6; c and d are each independently H, D, —OH, —OD, C1-C6-alkyl, —NH2 or —COCH3; and n is 1, 2, 3, 4 or 5.