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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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Dades bibliogràfiques
Publicat a:J Rare Dis Res Treat
Autors principals: Raja, Vaishnavi, Reynolds, Christian A., Greenberg, Miriam L.
Format: Artigo
Idioma:Inglês
Publicat: 2017
Matèries:
Accés en línia: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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