DOI: 10.1055/s-00029030

Journal of Pediatric Neurology

References

Sobrido MJ, Bauer P, de Koning T. et al.
Recommendations for patient screening in ultra-rare inherited metabolic diseases: what have we learned from Niemann-Pick disease type C?.

Orphanet J Rare Dis 2019;
14 (01) 20

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