IP Library › Patent Application 19074720
Patent Application
App. No. 19/074,720

METHODS AND COMPOSITIONS FOR THE PREVENTION OR TREATMENT OF BARTH SYNDROME

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Quick Facts
Patent No.
US None
App. No.
19/074,720
Abstract

The disclosure provides methods of preventing or treating Barth Syndrome in a mammalian subject, reducing risk factors associated with Barth Syndrome, and/or reducing the likelihood or severity of Barth Syndrome. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide to increase expression of TAZ1 in subjects in need thereof.

Claims (13)

1 .- 26 . (canceled)

27 . A method for improving LV function in a subject having Barth Syndrome, comprising administering to the subject a therapeutically effective amount of the peptide D-Arg-2′6′-Dmt-Lys-Phe-NH 2 or a pharmaceutically acceptable salt thereof.

28 . The method of claim 27 , wherein the peptide is administered daily for 6 weeks or more.

29 . The method of claim 27 , wherein the peptide is administered daily for 12 weeks or more.

30 . The method of claim 27 , wherein the subject is human.

31 . The method of claim 27 , wherein the peptide is administered orally, topically, systemically, intravenously, subcutaneously, intraperitoneally, or intramuscularly.

32 . The method of claim 27 , wherein the pharmaceutically acceptable salt comprises acetate or trifluoroacetate salt.

33 . A method for improving the rate of mitochondrial ATP synthesis in a subject having Barth Syndrome, comprising administering to the subject a therapeutically effective amount of the peptide D-Arg-2′6′-Dmt-Lys-Phe-NH 2 or a pharmaceutically acceptable salt thereof.

34 . The method of claim 33 , wherein the peptide is administered daily for 6 weeks or more.

35 . The method of claim 33 , wherein the peptide is administered daily for 12 weeks or more.

36 . The method of claim 33 , wherein the subject is human.

37 . The method of claim 33 , wherein the peptide is administered orally, topically, systemically, intravenously, subcutaneously, intraperitoneally, or intramuscularly.

38 . The method of claim 33 , wherein the pharmaceutically acceptable salt comprises acetate or trifluoroacetate salt.