Bibliografia Enfermedad de Niemann Pick C
Geberhiwot T, Moro A, Dardis A, Ramaswami U, Sirrs S et al.;
International Niemann-Pick Disease Registry (INPDR).
Consensus clinical management guidelines for Niemann-Pick disease type C.
Orphanet J Rare Dis. 2018 ;13:50
Bremova T, Malinová V, Amraoui Y, Mengel E, Reinke J et al.
Acetyl-dl-leucine in Niemann-Pick type C: A case series.
Neurology. 2015;85(16):1368-75
Pineda M, Juríčková K, Karimzadeh P, Kolnikova M, Malinova V et al.
Disease characteristics, prognosis and miglustat treatment effects on disease progression in patients with Niemann-Pick disease Type C: an international, multicenter, retrospective chart review.
Orphanet J Rare Dis. 2019;14:32
