Results from a 3-year Non-interventional, Observational Disease Monitoring Program in Adults with GNE Myopathy

BACKGROUND: GNE myopathy is a rare, autosomal recessive, muscle disease caused by mutations in GNE and is characterized by rimmed vacuoles on muscle biopsy and progressive distal to proximal muscle weakness. OBJECTIVE: Investigate the clinical presentation and progression of GNE myopathy. METHODS: T...

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Detalles Bibliográficos
Autores principales: Lochmüller, Hanns, Behin, Anthony, Tournev, Ivailo, Tarnopolsky, Mark, Horváth, Rita, Pogoryelova, Oksana, Shah, Jinay, Koutsoukos, Tony, Skrinar, Alison, Kakkis, Emil, Bedrosian, Camille L., Mozaffar, Tahseen
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2021
Materias:
Research Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8075380/
https://www.ncbi.nlm.nih.gov/pubmed/33459658
http://dx.doi.org/10.3233/JND-200565

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8075380/
https://www.ncbi.nlm.nih.gov/pubmed/33459658
http://dx.doi.org/10.3233/JND-200565

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