Treatment of friedreich's ataxia using histone deacetylase inhibitors
View Patent ↗The invention provides methods of treating Friedreich's ataxia and other neurodegenerative or neuromuscular conditions using histone deacetylase inhibitors.
1. 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;
R 1 is aryl or heteroaryl;
R 2 is aryl or heteroaryl;
R a and R b 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 NR c R d ;
wherein R c and R d are each independently hydrogen, alkyl, or C(═O)OR e wherein R e is H or alkyl;
or a salt thereof.
2. The method of claim 1 , further comprising identifying the mammal as one suffering from, or at risk for, Huntington's disease.
3. The method of claim 1 , wherein the mammal is a human.
4. The method of claim 1 , wherein the compound of formula I has a structure selected from the group consisting of:
a salt of a compound thereof; and
any combination thereof.