Journal article
Deferiprone in Friedreich ataxia: A 6‐Month randomized controlled trial
Abstract
OBJECTIVE: We conducted a 6-month, randomized, double-blind, placebo-controlled study to assess safety, tolerability, and efficacy of deferiprone in Friedreich ataxia (FRDA).
METHODS: Seventy-two patients were treated with deferiprone 20, 40, or 60mg/kg/day or placebo, divided into 2 daily doses. Safety was the primary objective; secondary objectives included standardized neurological assessments (Friedreich Ataxia Rating Scale [FARS], …
Authors
Pandolfo M; Arpa J; Delatycki MB; Le Quan Sang KH; Mariotti C; Munnich A; Sanz‐Gallego I; Tai G; Tarnopolsky MA; Taroni F
Journal
Annals of Neurology, Vol. 76, No. 4, pp. 509–521
Publisher
Wiley
Publication Date
October 2014
DOI
10.1002/ana.24248
ISSN
0364-5134