Participants of the 237th ENMC workshop which was held in June 2018, reviewed the current medical and scientific knowledge relevant to GNE myopathy to achieve a better understanding of GNE myopathy epidemiology, phenotype and genetics. They agreed on a standard of care (SOC) for GNE myopathy patients, discussed the strength and weakness of the currently available animal models, and attempted to improve their understanding of the biochemical consequences of the GNE defect on muscle tissue leading to muscle damage and weakness. Additionally, participants deliberated other potential treatments for the condition such as gene therapy and stem cell therapy.
A number of EURO-NMD network representatives not only attended the workshop but were invovled in the organisation of the workshop.
Meeting report – publication details
Upcoming events
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DMD Expert Masterclass
16 May 2019 -
12th Edition of the International Myotonic Dystrophy Consortium Meeting (IDMC-12)
10 June 2019 -
European Human Genetics Conference 2019
15 June 2019 -
9th World Congress on Rare Diseases and Orphan Drugs
17 June 2019 -
Summer School of Myology
17 June 2019
View complete list of events
