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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...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:JIMD Rep
Prif Awduron: Angelini, Corrado, Tavian, Daniela, Missaglia, Sara
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: Springer Berlin Heidelberg 2017
Pynciau:
Mynediad Ar-lein: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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