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https://www.selleckchem.com/products/dx3-213b.html Barth syndrome is a rare, life-threatening, X-linked recessive genetic disease that predominantly affects young males and is caused by abnormal mitochondrial lipid metabolism. Currently, there is no definitive treatment for Barth syndrome other than interventions to ameliorate acute symptoms, such as heart failure, cardiac arrhythmias, neutropenia, and severe muscle fatigue. Previous mechanistic studies have identified the lipid-lowering drug bezafibrate as a promising potential treatment; however, to date, no human trials have been performed in this population. The aim of this study is to determine whether bezafibrate (and resveratrol in vitro) will increase mitochondrial biogenesis and potentially modify the cellular ratio of monolysocardiolipin (MLCL) to tetralinoleoyl-cardiolipin (L4-CL), ameliorating the disease phenotype in those living with the disease. The CARDIOMAN (Cardiolipin Manipulation) study is a UK single-center, double-blinded, randomized, placebo-controlled crossover study investigatinp was completed in January 2020. Data analysis is ongoing, with publication expected in 2021. This trial was approved by the United Kingdom National Research Ethics Service Committee and the Medicines and Healthcare products Regulatory Agency. The feasibility of the CARDIOMAN study will help to inform the future conduct of randomized controlled trials in rare disease populations as well as testing the efficacy of bezafibrate as a potential treatment for the disease and advancing the mechanistic understanding of Barth syndrome. International Standard Randomized Controlled Trial Number (ISRCTN) 58006579; https//www.isrctn.com/ISRCTN58006579. DERR1-10.2196/22533. DERR1-10.2196/22533. Cognitive behavioral therapy (CBT) for bulimia nervosa (BN) is most effective when patients demonstrate adequate skill utilization (ie, the frequency with which a patient practices or uses therapeutic skills) and skill acquisition (ie, the a
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