Long-term evaluation parameters in GNE myopathy: a 5-year observational follow-up natural history study
Background A number of clinical trials targeting GNE myopathy patients have been conducted. However, useful clinical parameters for postmarketing surveillance and long-term clinical observation have not yet been established.Objective We conducted a 5-year observational follow-up natural history stud...
Main Authors: | , , , , , , |
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Format: | Article |
Language: | English |
Published: |
BMJ Publishing Group
2022-12-01
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Series: | BMJ Neurology Open |
Online Access: | https://neurologyopen.bmj.com/content/4/2/e000362.full |