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US 9,957,225 B2Granted
Treatment of friedreich's ataxia using histone deacetylase inhibitors
AssigneeThe Scripps Research Institute
InventorsJoel M. Gottesfeld, Ann-Kristin Jenssen, David M. Herman, Ryan Burnett, C. James Chou
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Abstract
The invention provides methods of treating Friedreich's ataxia and other neurodegenerative or neuromuscular conditions using histone deacetylase inhibitors.
Claims4
A method of treating, or delaying the onset of Huntington's disease in a mammal comprising administering to the mammal a compound of formula I in an amount effective to alter the level of histone acetylation in the mammal and increase frataxin mRNA in lymphocytes from Friedreich's ataxia patients, wherein formula I is:
- wherein:
- n is 2 to about 10;
- R1 is aryl or heteroaryl;
- R2 is aryl or heteroaryl;
- Ra and Rb are each independently H, alkyl, aryl, heteroaryl, or a nitrogen protecting group;
- wherein any alkyl, aryl or heteroaryl is optionally substituted with 1 to 3 substituents selected from the group consisting of hydroxy, amino, nitro, cyano, halo, alkyl, trifluoromethyl, alkoxy, aryl, and NRcRd;
- wherein Rc and Rd are each independently hydrogen, alkyl, or C(═O)ORe wherein Re is H or alkyl;
- or a salt thereof.
5 more elements
Dependencies are inferred from the claim text.