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Heterogeneous Phenotypes in Lipid Storage Myopathy Due to ETFDH Gene Mutations

We present six novel patients affected by lipid storage myopathy (LSM) presenting mutations in the ETFDH gene. Although the diagnosis of multiple acyl-coenzyme-A dehydrogenase deficiency (MADD) in adult life is difficult, it is rewarding because of the possibility of treating patients with carnitine...

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Publicat a:JIMD Rep
Autors principals: Angelini, Corrado, Tavian, Daniela, Missaglia, Sara
Format: Artigo
Idioma:Inglês
Publicat: Springer Berlin Heidelberg 2017
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5874208/
https://ncbi.nlm.nih.gov/pubmed/28456887
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/8904_2017_27
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