Niemann-Pick B - RS - 5th infusion.

Interstitial lung disease in lysosomal storage disorders

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)

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Promoter considerations in the design of lentiviral vectors for use in treating lysosomal storage diseases: Molecular Therapy - Methods & Clinical Development

Natural history and management of liver dysfunction in lysosomal storage disorders

Pulmonary Type B Niemann-Pick Disease Successfully Treated with Lung Transplantation

Niemann–Pick disease - Wikipedia

Miglustat in Niemann-Pick disease type C patients: a review, Orphanet Journal of Rare Diseases

Frontiers Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases

Splenomegaly, hypersplenism, and hereditary disorders with splenomegaly

Sphingomyelin-induced inhibition of the plasma membrane calcium ATPase causes neurodegeneration in type A Niemann–Pick disease

(PDF) Niemann-Pick Disease: An Underdiagnosed Lysosomal Storage Disorder

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PDF) Chronic visceral acid sphingomyelinase deficiency (Niemann-Pick disease type B) in 16 Polish patients: Long-term follow-up