Results 131 to 138 of about 406 (138)

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)

Genetics in Medicine, 2016
Melissa P Wasserstein
exaly  

Elastoresonance as a surveillance tool in a patient with acid sphingomyelinase deficiency undergoing treatment with olipudase alfa

Molecular Genetics and Metabolism
Magdalena Ceron-Rodriguez, Carlos P. Acosta Rodríguez Bueno, Judith Almanza-Aranda, Alan E. Araujo Carrillo, Karla Cifuentes-Uribe   +4 more
openaire   +1 more source

Quantitative systems pharmacology model of acid sphingomyelinase deficiency and the enzyme replacement therapy olipudase alfa is aninnovative tool for linking pathophysiology and pharmacology

Molecular Genetics and Metabolism, 2017
Chanchala Kaddi, Rena Baek, Paul Jasper, Bradley Niesner, John Pappas, John Tolsma, Jing Li, Shayne Watson, Sharon Tan, Ana Cristina Puga, Edward Schuchman, Jeffrey S. Barrett, Karim Azer   +12 more
openaire   +1 more source

Novel Case of Olipudase Alfa Enzyme Replacement Therapy to Treat Niemann-Pick Disease Type-B Following Lung Transplant

The Journal of Heart and Lung Transplantation
J. Ng, G.F. Watts, M. Musk, J. Wrobel, M. Lavender, S. Lawrence, C.J. Welman, C. Wood, J. Polce, M. Yaw   +9 more
openaire   +1 more source

Olipudase alfa enzyme replacement therapy reverses interstitial lung disease in adults with acid sphingomyelinase deficiency: Long-term pulmonary outcomes of the ASCEND trial

Molecular Genetics and Metabolism
Wasserstein, Melissa, Bonella, Francesco, Giugliani, Roberto, Lachmann, Robin, Noth, Imre, Scarpa, Maurizio, Wuyts, Wim A., Aguiar, Mario, Armstrong, Nicole, Kumar, Monica, Berger, Kenneth   +10 more
openaire   +1 more source

Reversal of Interstitial Lung Disease in Children and Adults With Chronic Acid Sphingomyelinase Deficiency Treated With Olipudase Alfa Enzyme Replacement Therapy

American Journal of Respiratory and Critical Care Medicine
G. Raghu, M. Scarpa, R. Giugliani, R. Lachmann, M. Wasserstein, F. Bonella, I. Noth, W.A. Wuyts, N.M. Armstrong, M. Kumar, K.I. Berger   +10 more
openaire   +1 more source

Peginterferon alfa-2b plus ribavirin compared with interferon alfa-2b plus ribavirin for initial treatment of chronic hepatitis C: a randomised trial

Lancet, The, 2001
exaly  

On the role of excipients in biopharmaceuticals manufacture: Modelling-guided formulation identifies the protective effect of arginine hydrochloride excipient on spray-dried Olipudase alfa recombinant protein

International Journal of Pharmaceutics
Ashutosh Sharma, Damien Thompson, Helen Hughes   +2 more
exaly