DOI:
10.1055/s-00029022
Journal of Pediatric Biochemistry
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References
Patterson MC, Mengel E, Vanier MT , et al; NPC Registry investigators.
Stable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: an observational cohort study.
Orphanet J Rare Dis 2015;
10: 65
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