Niemann-Pick B - RS - 5th infusion.
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Biochemical and imaging parameters in acid sphingomyelinase deficiency: Potential utility as biomarkers - ScienceDirect
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Figure 3 from Niemann-Pick disease type B. Study of 3 cases and literature revision
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Niemann-Pick disease type C
Olipudase alfa enzyme replacement therapy for acid sphingomyelinase deficiency (ASMD): sustained improvements in clinical outcomes after 6.5 years of treatment in adults, Orphanet Journal of Rare Diseases
Bilateral Cystic Bronchiectasis as Novel Phenotype of Niemann-Pick Disease Type B Successfully Treated With Double Lung Transplantation - CHEST
Niemann–Pick disease - Wikipedia
PDF) Novel first-dose adverse drug reactions during a phase I trial of olipudase alfa (recombinant human acid sphingomyelinase) in adults with Niemann–Pick disease type B (acid sphingomyelinase deficiency)
Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial - The Lancet
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