Methods of treatment for mitochondrial DNA depletion disorders

Abstract

The present invention provides a method for treating a subject suffering from primary mitochondrial disease (PMD) or mitochondrial DNA depletion syndrome (MDDS), the method comprising administering a therapeutically effective amount of a compound or a pharmaceutically acceptable salt thereof listed in Table 1, Table 2, Table 3, Table 4 or Table 5, wherein the subject does not have a mutation in the DNA polymerase γ gene (POLG). Provided are methods of treating a subject having a Primary Mitochondrial Disorder (PMD) or Mitochondrial DNA Depletion Syndrome (MDDS) comprising administering a therapeutically effective amount of a compound listed in Table 1, Table 2, Table 3, Table 4, or Table 5, or a pharmaceutically acceptable salt thereof, wherein the subject does not have a mutation in the DNA polymerase γ gene (POLG).

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