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Methods and compositions for the prevention or treatment of Barth syndrome
Methods and compositions for the prevention or treatment of Barth syndrome
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机译:预防或治疗巴氏综合征的方法和组合物
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摘要
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.
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