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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...

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Detalles Bibliográficos
Autores principales:	Mori-Yoshimura, Madoka, Yajima, Hiroyuki, Oya, Yasushi, Mizuno, Katsuhiro, Noguchi, Satoru, Nishino, Ichizo, Takahashi, Yuji
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BMJ Publishing Group 2022
Materias:	Original Research
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

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