Prodrugs of deoxynucleosides for treatment of mitochondrial diseases caused by unbalanced nucleotide pools
AssigneeUcb Biosciences, Inc.
InventorDaniel DiPIETRO
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Abstract
Deoxynucleotide prodrugs and methods of their use for treatment of diseases characterized by unbalanced nucleotide pools such as MPV17 and deoxyguanosine kinase deficiency are provided herein.
Claims22
-17. (Canceled)
A method for treating a mitochondrial DNA depletion disease or disorder in a subject in need thereof comprising administering to the subject a therapeutically effective amount of at least one first prodrug of formula:
- wherein R is selected from the group consisting of cytosine, thymine, guanine, or adenine.
A method for treating a mitochondrial depletion disease or disorder in a subject in need thereof comprising administering to the subject a therapeutically effective amount of at least one prodrug of formula:
- wherein R is selected from the group consisting of cytosine, thymine, guanine, or adenine.
A method of monitoring an improvement in a condition of a subject after administration to the subject a therapeutically effective amount of at least one first prodrug of formula:
- wherein R is selected from the group consisting of cytosine, thymine, guanine, or adenine;
- and wherein an improvement in the condition of the subject is exhibited by an increase in muscle strength, an increase in muscle control, an increase in mobility of the subject, an increase in subject height, and/or an increase in subject weight.
Dependencies are inferred from the claim text.