IP Library Granted Patent US 9,957,225
Granted Patent B2
US 9,957,225 · App. 14/480,899 · Granted May 1, 2018

Treatment of friedreich's ataxia using histone deacetylase inhibitors

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Quick Facts
Patent No.
US 9,957,225
App. No.
14/480,899
Granted
May 1, 2018
Kind
B2
Abstract

The invention provides methods of treating Friedreich's ataxia and other neurodegenerative or neuromuscular conditions using histone deacetylase inhibitors.

Claims (14)

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.