4-(p-quinonyl)-2-hydroxybutanamide derivatives for treatment...

C - Chemistry – Metallurgy – 07 – C

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Details

C07C 235/78 (2006.01) A61K 31/16 (2006.01) A61K 31/445 (2006.01) A61P 25/00 (2006.01) A61P 25/16 (2006.01) C07C 317/28 (2006.01) C07D 207/27 (2006.01) C07D 213/40 (2006.01) C07D 223/04 (2006.01) C07D 233/61 (2006.01) C07D 295/02 (2006.01) C07D 295/185 (2006.01) C07D 295/192 (2006.01) C07D 295/24 (2006.01) C07D 307/14 (2006.01)

Patent

CA 2704473

Methods of treating or suppressing mitochondrial diseases, such as Friedreich's ataxia (FRDA), Leber's Hereditary Optic Neuropathy (LHON), mitochondrial myopathy, encephalopathy, lactacidosis, and stroke (MELAS), Kearns-Sayre Syndrome (KSS), are disclosed, as well as compounds useful in the methods of the invention, such as 4-(p- quinolyl)-2-hydroxybutanamide derivatives. Methods and compounds useful in treating other disorders such as amyotrophic lateral sclerosis (ALS), Huntington's disease, Parkinson's disease, and pervasive developmental disorders such as autism are also disclosed. Energy biomarkers useful in assessing the metabolic state of a subject and the efficacy of treatment are also disclosed. Methods of modulating, normalizing, or enhancing energy biomarkers, as well as compounds useful for such methods, are also disclosed.

L'invention concerne des procédés de traitement ou de suppression de maladies mitochondriales, comme l'ataxie de Friedreich (FRDA), la neuropathie optique héréditaire de Leber (LHON), la myopathie congénitale mitochondriale, l'encéphalopathie, l'acidose lactique, les accidents vasculaires cérébraux (MELAS)et le syndrome de Kearns-Sayre (KSS), ainsi que des composés qui s'utilisent dans les procédés de l'invention, tels des dérivés de 4-(p-quinolyl)-2-hydroxybutanamide. L'invention concerne également des procédés et des composés qui s'utilisent dans le traitement d'autres troubles tels que la sclérose amyotrophique latérale (ALS), la maladie de Huntington, la maladie de Parkinson et des troubles envahissants du développement tels que l'autisme. L'invention concerne aussi des biomarqueurs d'énergie qui s'utilisent dans l'évaluation de l'état métabolique du sujet et de l'efficacité du traitement. Des procédés de modulation, de normalisation ou d'amélioration de biomarqueurs d'énergie, ainsi que des composés qui s'utilisent dans de tels procédés, sont également décrits.

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