Niemann-Pick B - RS - 5th infusion.

Acid Sphingomyelinase Overexpression Enhances the Antineoplastic Effects of Irradiation In Vitro and In Vivo: Molecular Therapy

Interstitial lung disease in lysosomal storage disorders

Frontiers Expert opinion on patient journey, diagnosis and clinical monitoring in acid sphingomyelinase deficiency in Turkey: a pediatric metabolic disease specialist's perspective

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

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

JCM, Free Full-Text

PDF) Changes in PCSK 9 and Apolipoprotein B100 in Niemann-Pick Disease After Enzyme Replacement Therapy with Olipudase Alfa

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

New Treatment of Disease that Swells Organs Gives New Hope to 4-year-old Girl - International Niemann-Pick Disease Alliance (INPDA)

Potential treatment for Niemann-Pick type C, a rare neurodegenerative disease

Lysosomal storage disorders: from biology to the clinic with reference to India - ScienceDirect

British Journal of Haematology

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)

Niemann-Pick disease type C

Investigational New Drug (IND) - The Addi and Cassi Fund