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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 s...
Autores principales: | , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BMJ Publishing Group
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9723845/ https://www.ncbi.nlm.nih.gov/pubmed/36483092 http://dx.doi.org/10.1136/bmjno-2022-000362 |