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Phenotypic stratification and genotype–phenotype correlation in a heterogeneous, international cohort of GNE myopathy patients: First report from the GNE myopathy Disease Monitoring Program, registry portion

GNE myopathy is a rare distal myopathy, caused by mutations in the GNE gene, affecting sialic acid synthesis. Clinical presentation varies from asymptomatic early stage patients to severely debilitating forms. This first report describes clinical presentations and severity of the disease, using data...

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Bibliografski detalji
Izdano u:Neuromuscul Disord
Glavni autori: Pogoryelova, Oksana, Cammish, Phillip, Mansbach, Hank, Argov, Zohar, Nishino, Ichizo, Skrinar, Alison, Chan, Yiumo, Nafissi, Shahriar, Shamshiri, Hosein, Kakkis, Emil, Lochmüller, Hanns
Format: Artigo
Jezik:Inglês
Izdano: Pergamon Press 2018
Teme:
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5857291/
https://ncbi.nlm.nih.gov/pubmed/29305133
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.nmd.2017.11.001
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