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

BACKGROUND: 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 symp...

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Dades bibliogràfiques
Publicat a:JMIR Res Protoc
Autors principals: Dabner, Lucy, Pieles, Guido E, Steward, Colin G, Hamilton-Shield, Julian P, Ness, Andrew R, Rogers, Chris A, Bucciarelli-Ducci, Chiara, Greenwood, Rosemary, Ellis, Lucy, Sheehan, Karen, Reeves, Barnaby C
Format: Artigo
Idioma:Inglês
Publicat: JMIR Publications 2021
Matèries:
Protocol
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC8204243/
https://ncbi.nlm.nih.gov/pubmed/34057417
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2196/22533
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