Article

Long-term Evaluation Parameters in GNE Myopathy: A Five-year Observational Follow-up Natural History Study

2021-10-13

Abstract excerpt

<title>Abstract</title> <p>BackgroundA number of clinical trials targeting GNE myopathy patients have been conducted. However, useful clinical parameters for post-marketing surveillance and long-term clinical observation have not yet been established. ObjectiveWe conducted a 5-year observational follow-up natural history study to identify evaluation parameters which may be useful for the long-term observation of...

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Literature Corpus work: d2548093-e117-5789-adb0-957a2776e0b9
DOI: 10.21203/rs.3.rs-909985/v1

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