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)

Nonclinical safety assessment of recombinant human acid sphingomyelinase (rhASM) for the treatment of acid sphingomyelinase deficiency: The utility of animal models of disease in the toxicological evaluation of potential therapeutics

(2015) James M. Murray et al.   MOLECULAR GENETICS AND METABOLISM

Treatment of lysosomal storage disorders: successes and challenges

(2014) Carla E. M. Hollak et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Morbidity and mortality in type B Niemann–Pick disease

(2013) Margaret M. McGovern et al.   GENETICS IN MEDICINE

Liver and Skin Histopathology in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease Type B)

(2012) Beth L. Thurberg et al.   AMERICAN JOURNAL OF SURGICAL PATHOLOGY

Transplante hepático em paciente portadora de doença de Niemann-Pick com envolvimento pulmonar

(2012) Marina Silveira Mendes et al.   Jornal Brasileiro de Pneumologia

Characterization of and Risk Factors for the Acute-Phase Response after Zoledronic Acid

(2010) I. R. Reid et al.   JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM

Gilbert-Meulengracht’s syndrome and pharmacogenetics: is jaundice just the tip of the iceberg?

(2009) Christian P. Strassburg   DRUG METABOLISM REVIEWS

A Prospective, Cross-sectional Survey Study of the Natural History of Niemann-Pick Disease Type B

(2008) M. M. McGovern et al.   PEDIATRICS