Approximately 5‐year difference in median survival between the two analysis groups may be reflective of the observed 4.29‐year mean delay between the onset of neurological manifestations and NP‐C diagnosis, with the KM curves also demonstrating a clear trend for longer survival with miglustat treatment.
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Long-term survival outcomes of patients with Niemann-Pick disease type C receiving miglustat treatment: A large retrospective observational study.
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