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...

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Bibliographic Details
Main Authors: Yuji Takahashi, Ichizo Nishino, Madoka Mori-Yoshimura, Yasushi Oya, Satoru Noguchi, Hiroyuki Yajima, Katsuhiro Mizuno
Format: Article
Language:English
Published: BMJ Publishing Group 2022-12-01
Series:BMJ Neurology Open
Online Access:https://neurologyopen.bmj.com/content/4/2/e000362.full