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Barth syndrome: A life-threatening disorder caused by abnormal cardiolipin remodeling

Barth syndrome (BTHS) is a rare X-linked genetic disorder characterized by cardiomyopathy, skeletal myopathy, neutropenia, and organic aciduria. The presence and severity of clinical manifestations are highly variable in BTHS, even among patients with identical gene mutations. Currently, less than 2...

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Bibliographische Detailangaben
Veröffentlicht in:J Rare Dis Res Treat
Hauptverfasser: Raja, Vaishnavi, Reynolds, Christian A., Greenberg, Miriam L.
Format: Artigo
Sprache:Inglês
Veröffentlicht: 2017
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Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6482962/
https://ncbi.nlm.nih.gov/pubmed/31032491
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.29245/2572-9411/2017/2.1087
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