CAMBRIDGE, Mass.--(BUSINESS WIRE)--FoldRx Pharmaceuticals, Inc. (FoldRx) today announced that enrollment is underway in a multinational Phase II/III clinical study with its lead drug candidate, Fx-1006A, for patients suffering from Familial Amyloid Polyneuropathy (FAP), a rare genetic disease that affects approximately 10,000 people worldwide. FAP is a fatal disorder in which amyloid fibrils, caused by the ‘misfolding’ of a protein called transthyretin (TTR), are deposited in peripheral nerve tissues that serve the limbs and organs. FAP results in progressive loss of sensation and motor function, organ failure, pain and weakness. Fx-1006A is designed to stop the progression of this disease by preventing the accumulation of amyloid fibrils caused by the ‘misfolding’ of TTR. Protein folding is the process by which a protein assumes its characteristic functional shape and is essential for normal cellular function.
