DEFERIPRONE FOR USE IN TREATING FRIEDREICH ATAXIA RESULTING FROM MITOCHONDRIAL IRON INDUCED DAMAGE IN THE ABSENCE OF GENERALISED IRON OVERLOAD

摘要

A therapeutically effective amount of deferiprone or deferasirox or physiologically acceptable salts thereof for the prevention, stabilization, treatment, or reversal of iron-induced FRDA disease in patients resulting from mitochondrial iron-induced damage to preferentially reduce the iron stores in the mitochondria. Also for the treatment of other conditions affecting the brain where a key element in the generation of the resultant pathology is the intracellular mishandling of iron.