DOI: 10.1055/s-00029022

Journal of Pediatric Biochemistry

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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