Methods and compositions for the treatment of sengers syndrome
AssigneeStealth BioTherapeutics Inc.
InventorsJames Carr, Michael Dimatteo, Anthony Abbruscato, Hans Bjornsson
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Abstract
The disclosure provides methods of preventing or treating Sengers syndrome in a mammalian subject, reducing risk factors associated with Sengers syndrome, and/or reducing the likelihood or severity of Sengers syndrome. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide to increase expression of AGK in subjects in need thereof.
Claims24
A method for treating or preventing Sengers syndrome in a subject in need thereof, comprising administering to the subject a therapeutically effective amount of the peptide D-Arg-2′6′-Dmt-Lys-Phe-NH2 or a pharmaceutically acceptable salt thereof.
A method for increasing the expression of AGK in a mammalian subject in need thereof, the method comprising:
- administering to the subject a therapeutically effective amount of the peptide D-Arg-2′6′-Dmt-Lys-Phe-NH2 or a pharmaceutically acceptable salt thereof.
A method for reducing the risk of Sengers syndrome in a mammalian subject having decreased expression of AGK compared to a normal control subject, the method comprising:
- administering to the subject a therapeutically effective amount of the peptide D-Arg-2′6′-Dmt-Lys-Phe-NH2 or a pharmaceutically acceptable salt thereof.
Dependencies are inferred from the claim text.