Methods, compositions, and uses related to the treatment and prevention of leigh syndrome
AssigneeStealth BioTherapeutics Inc.
InventorsDavid Brown, Hatim Zariwala, Laura Kropp, Alyssa Handler
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Abstract
The present disclosure provides methods for treating, preventing, ameliorates, inhibits, or delaying the onset of Leigh syndrome as well as methods for ameliorating, inhibiting, ameliorates, inhibits, or delaying the onset of its associated signs and/or symptoms, such as exercise-induced acid acidosis, and increases in plasma levels of growth factor 15 (GDF-15) and/or fibroblast growth factor (FGF-21) in a subject in need thereof comprising administering to the subject various compounds, mixtures of compounds or compositions, formulations or medicaments derived therefrom.
Claims20
A method for preventing, inhibiting or delaying the onset of exercise-induced lactic acidosis in a subject in need thereof, comprising administering to the subject a therapeutically effective amount of Compound 1, Compound 2, Compound 3, Compound 4, or a mixture of any two or more of Compounds 1 to 4, or pharmaceutically acceptable salts, stereoisomers, mixtures of stereoisomers, tautomers, hydrates, and/or solvates thereof, wherein Compounds 1 to 4 have the following structures:
- and wherein the subject has been diagnosed with or is suspected of having Leigh syndrome.
A method for attenuating an increase in plasma levels of growth differentiation factor 15 (GDF-15) and/or fibroblast growth factor 21 (FGF-21) in a subject in need thereof, comprising administering to the subject a therapeutically effective amount of Compound 1, Compound 2, Compound 3, Compound 4, or a mixture of any two or more of Compounds 1 to 4, or pharmaceutically acceptable salts, stereoisomers, mixtures of stereoisomers, tautomers, hydrates, and/or solvates thereof, wherein Compounds 1 to 4 have the following structures:
- and wherein the subject has been diagnosed with or is suspected of having Leigh syndrome.
Dependencies are inferred from the claim text.