Use of deferiprone and methods to treat and/or prevent friedreich ataxia resulting from intracellular mishandling of iron
OwnerArnold Munnich, Michael Spino +1inventors
InventorsArnold Munnich, Michael Spino, Ioav Cabantchik
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Abstract
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.
Claims28
-29. (canceled)
A method of treating Friedreich ataxia in patients resulting from mitochondrial iron-induced damage, comprising administering to the patient a therapeutically effective amount of deferiprone, deferasirox or physiologically acceptable salts thereof sufficient to treat Friedriech ataxia resulting from mitochondrial iron-induced damage.
A method of preventing the development of symptoms of Friedreich ataxia in patients resulting from mitochondrial iron-induced damage, comprising administering to the patient a therapeutically effective amount of deferiprone, deferasirox or physiologically acceptable salts thereof sufficient to prevent symptoms of Friedreich ataxia.
A method to reduce or render inactive the toxic iron stores in the subcellular compartments of the brain, and to remove iron from these compartments, and/or to mitigate the cellular or intracellular mishandling of iron in the brain comprising the administration of a sufficient amount of a therapeutically effective amount of a cell penetrant oral iron chelator, such as deferiprone, deferasirox, or physiologically acceptable salts thereof.
Dependencies are inferred from the claim text.