Claims for Patent: 12,268,724

Summary for Patent: 12,268,724

Title:	Methods and compositions for the prevention or treatment of Barth Syndrome
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.
Inventor(s):	D. Travis Wilson, Mark Bamberger
Assignee:	Stealth Biotherapeutics Inc
Application Number:	US18/454,319
Patent Claims:	1. A method for reducing the number of mitochondria with abnormal mitochondrial ultrastructure, ameliorating abnormal mitochondrial ultrastructure, or maintaining mitochondrial organization 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-NH2 or a pharmaceutically acceptable salt thereof. 2. The method of claim 1, wherein the peptide is administered daily for 6 weeks or more. 3. The method of claim 1, wherein the peptide is administered daily for 12 weeks or more. 4. The method of claim 1, wherein the subject is human. 5. The method of claim 1, wherein the peptide is administered orally, topically, systemically, intravenously, subcutaneously, intraperitoneally, or intramuscularly. 6. The method of claim 1, further comprising separately, sequentially or simultaneously administering a cardiovascular agent to the subject. 7. The method of claim 6, wherein the cardiovascular agent is selected from the group consisting of: an anti-arrhythmia agent, a vasodilator, an anti-anginal agent, a corticosteroid, a cardioglycoside, a diuretic, a sedative, an angiotensin converting enzyme (ACE) inhibitor, an angiotensin II antagonist, a thrombolytic agent, a calcium channel blocker, a throboxane receptor antagonist, a radical scavenger, an anti-platelet drug, a β-adrenaline receptor blocking drug, an α-receptor blocking drug, a sympathetic nerve inhibitor, a digitalis formulation, an inotrope, and an antihyperlipidemic drug. 8. The method of claim 1, wherein the pharmaceutically acceptable salt comprises acetate or trifluoroacetate salt.