This paper is in the following e-collection/theme issue:

RCTs - Protocols/Proposals (funded, already peer-reviewed, non-eHealth) (172) Cardiac Disease Management (94) Pharmacology and Pharmaceutics (66) Drug Repurposing and Off-Label Use (25) Chronic Conditions (270)

Published on in Vol 10, No 5 (2021): May

Preprints (earlier versions) of this paper are available at https://preprints.jmir.org/preprint/22533, first published .
Treatment of Barth Syndrome by Cardiolipin Manipulation (CARDIOMAN) With Bezafibrate: Protocol for a Randomized Placebo-Controlled Pilot Trial Conducted in the Nationally Commissioned Barth Syndrome Service

Treatment of Barth Syndrome by Cardiolipin Manipulation (CARDIOMAN) With Bezafibrate: Protocol for a Randomized Placebo-Controlled Pilot Trial Conducted in the Nationally Commissioned Barth Syndrome Service

Treatment of Barth Syndrome by Cardiolipin Manipulation (CARDIOMAN) With Bezafibrate: Protocol for a Randomized Placebo-Controlled Pilot Trial Conducted in the Nationally Commissioned Barth Syndrome Service

Authors of this article:

Lucy Dabner1 Author Orcid Image ;   Guido E Pieles2, 3 Author Orcid Image ;   Colin G Steward4 Author Orcid Image ;   Julian P Hamilton-Shield5 Author Orcid Image ;   Andrew R Ness5, 6 Author Orcid Image ;   Chris A Rogers1 Author Orcid Image ;   Chiara Bucciarelli-Ducci3, 7 Author Orcid Image ;   Rosemary Greenwood8 Author Orcid Image ;   Lucy Ellis1 Author Orcid Image ;   Karen Sheehan9 Author Orcid Image ;   Barnaby C Reeves1 Author Orcid Image
Article Authors Cited by (13) Tweetations (4) Metrics

Journals

  1. Płatek T, Sordyl M, Polus A, Olszanecka A, Kroczka S, Solnica B. Analysis of tafazzin and deoxyribonuclease 1 like 1 transcripts and X chromosome sequencing in the evaluation of the effect of mosaicism in the TAZ gene on phenotypes in a family affected by Barth syndrome. Mutation Research/Fundamental and Molecular Mechanisms of Mutagenesis 2023;826:111812 View
  2. Greenwell A, Tabatabaei Dakhili S, Ussher J. Myocardial disturbances of intermediary metabolism in Barth syndrome. Frontiers in Cardiovascular Medicine 2022;9 View
  3. Chan J, Fernandes M, Steckel K, Bradley R, Hashemi A, Groh M, Sciaini G, Stark K, Duncan R. N-oleoylethanolamide treatment of lymphoblasts deficient in Tafazzin improves cell growth and mitochondrial morphology and dynamics. Scientific Reports 2022;12(1) View
  4. da Rosa-Junior N, Parmeggiani B, Glänzel N, de Moura Alvorcem L, Brondani M, Britto R, Grings M, Ortiz V, Turck P, da Rosa Araujo A, Wajner M, Leipnitz G. Antioxidant system disturbances and mitochondrial dysfunction induced by 3-methyglutaric acid in rat heart are prevented by bezafibrate. European Journal of Pharmacology 2022;924:174950 View
  5. Tragni V, Primiano G, Tummolo A, Cafferati Beltrame L, La Piana G, Sgobba M, Cavalluzzi M, Paterno G, Gorgoglione R, Volpicella M, Guerra L, Marzulli D, Servidei S, De Grassi A, Petrosillo G, Lentini G, Pierri C. Personalized Medicine in Mitochondrial Health and Disease: Molecular Basis of Therapeutic Approaches Based on Nutritional Supplements and Their Analogs. Molecules 2022;27(11):3494 View
  6. Greenwell A, Tabatabaei Dakhili S, Gopal K, Saed C, Chan J, Kazungu Mugabo N, Zhabyeyev P, Eaton F, Kruger J, Oudit G, Ussher J. Stimulating myocardial pyruvate dehydrogenase activity fails to alleviate cardiac abnormalities in a mouse model of human Barth syndrome. Frontiers in Cardiovascular Medicine 2022;9 View
  7. Pang J, Bao Y, Mitchell-Silbaugh K, Veevers J, Fang X. Barth Syndrome Cardiomyopathy: An Update. Genes 2022;13(4):656 View
  8. Thompson R, Jefferies J, Wang S, Pu W, Takemoto C, Hornby B, Heyman A, Chin M, Vernon H. Current and future treatment approaches for Barth syndrome. Journal of Inherited Metabolic Disease 2022;45(1):17 View
  9. Richardson K, Wessells R, Messina G. A novel panel of Drosophila TAFAZZIN mutants in distinct genetic backgrounds as a resource for therapeutic testing. PLOS ONE 2023;18(9):e0286380 View
  10. Joshi A, Richard T, Gohil V. Mitochondrial phospholipid metabolism in health and disease. Journal of Cell Science 2023;136(17) View
  11. Dale D, Bolyard A, Makaryan V. The promise of novel treatments for severe chronic neutropenia. Expert Review of Hematology 2023;16(12):1025 View
  12. Kagan V, Tyurina Y, Mikulska-Ruminska K, Damschroder D, Vieira Neto E, Lasorsa A, Kapralov A, Tyurin V, Amoscato A, Samovich S, Souryavong A, Dar H, Ramim A, Liang Z, Lazcano P, Ji J, Schmidtke M, Kiselyov K, Korkmaz A, Vladimirov G, Artyukhova M, Rampratap P, Cole L, Niyatie A, Baker E, Peterson J, Hatch G, Atkinson J, Vockley J, Kühn B, Wessells R, van der Wel P, Bahar I, Bayir H, Greenberg M. Anomalous peroxidase activity of cytochrome c is the primary pathogenic target in Barth syndrome. Nature Metabolism 2023;5(12):2184 View
  13. Pieles G, Steward C, Dabner L, Collet L, Culliford L, Sheehan K, Ellis L, Damin M, Sammut E, Duarte N, Burgess O, Wadey C, Williams C, Crosby J, Groves S, Searle A, Amulic B, Rice C, Bucciarelli-Ducci C, Ness A, Hamilton-Shield J, Rogers C, Reeves B. Bezafibrate as treatment in males for Barth syndrome: CARDIOMAN, a double-blind, placebo-controlled crossover RCT. Efficacy and Mechanism Evaluation 2024:1 View