Long-term treatment effect in cerebrotendinous xanthomatosis depends on age at treatment start.
Stelten BML, Huidekoper HH, van de Warrenburg BPC, Brilstra EH, Hollak CEM, Haak HR, Kluijtmans LAJ, Wevers RA, Verrips A
Neurology, 2019 01 08
Abstract
To evaluate the effect of chenodeoxycholic acid treatment on disease progression in cerebrotendinous xanthomatosis (CTX).