Cargando…

Quantitative Systems Pharmacology Modeling of Acid Sphingomyelinase Deficiency and the Enzyme Replacement Therapy Olipudase Alfa Is an Innovative Tool for Linking Pathophysiology and Pharmacology

Acid sphingomyelinase deficiency (ASMD) is a rare lysosomal storage disorder with heterogeneous clinical manifestations, including hepatosplenomegaly and infiltrative pulmonary disease, and is associated with significant morbidity and mortality. Olipudase alfa (recombinant human acid sphingomyelinas...

Descripción completa

Detalles Bibliográficos
Autores principales:	Kaddi, Chanchala D., Niesner, Bradley, Baek, Rena, Jasper, Paul, Pappas, John, Tolsma, John, Li, Jing, van Rijn, Zachary, Tao, Mengdi, Ortemann‐Renon, Catherine, Easton, Rachael, Tan, Sharon, Puga, Ana Cristina, Schuchman, Edward H., Barrett, Jeffrey S., Azer, Karim
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2018
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6063739/ https://www.ncbi.nlm.nih.gov/pubmed/29920993 http://dx.doi.org/10.1002/psp4.12304

Ejemplares similares

One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency
por: Diaz, George A., et al.
Publicado: (2021)

One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency
por: Diaz, George A., et al.
Publicado: (2022)

Long-term safety and clinical outcomes of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency: two-year results
por: Diaz, George A., et al.
Publicado: (2022)

Correction : Long-term safety and clinical outcomes of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency: two-year results
por: Diaz, George A., et al.
Publicado: (2023)

Clearance of Hepatic Sphingomyelin by Olipudase Alfa Is Associated With Improvement in Lipid Profiles in Acid Sphingomyelinase Deficiency
por: Thurberg, Beth L., et al.
Publicado: (2016)

A Quantitative Systems Pharmacology Model of Gaucher Disease Type 1 Provides Mechanistic Insight Into the Response to Substrate Reduction Therapy With Eliglustat
por: Abrams, Ruth, et al.
Publicado: (2020)

Olipudase alfa for treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months
por: Wasserstein, Melissa P., et al.
Publicado: (2018)

Olipudase Alfa in Non-CNS Manifestations of Acid Sphingomyelinase Deficiency: A Profile of Its Use
por: Syed, Yahiya Y.
Publicado: (2023)

PB2314: OLIPUDASE ALFA FOR ADULTS WITH ACID SPHINGOMYELINASE DEFICIENCY: IMPROVEMENTS IN CROSSOVER PLACEBO PATIENTS AND FURTHER IMPROVEMENTS IN ORIGINAL OLIPUDASE ALFA PATIENTS AFTER 2 YEARS IN ASCEND TRIAL
por: Villarrubia, J., et al.
Publicado: (2022)

Correction to: Olipudase Alfa in Non-CNS Manifestations of Acid Sphingomyelinase Deficiency: A Profile of Its Use
por: Syed, Yahiya Y.
Publicado: (2023)

Olipudase alfa enzyme replacement therapy for acid sphingomyelinase deficiency (ASMD): sustained improvements in clinical outcomes after 6.5 years of treatment in adults
por: Lachmann, Robin H., et al.
Publicado: (2023)

Continued improvement in disease manifestations of acid sphingomyelinase deficiency for adults with up to 2 years of olipudase alfa treatment: open-label extension of the ASCEND trial
por: Wasserstein, Melissa P., et al.
Publicado: (2023)

P1483: LONG-TERM IMPACT OF OLIPUDASE ALFA ENZYME REPLACEMENT THERAPY ON SPLEEN VOLUME AND HEMATOLOGIC MANIFESTATIONS IN CHILDREN AND ADULTS WITH CHRONIC ACID SPHINGOMYELINASE DEFICIENCY
por: Villarrubia, Jesus, et al.
Publicado: (2023)

History and Future Perspectives on the Discipline of Quantitative Systems Pharmacology Modeling and Its Applications
por: Azer, Karim, et al.
Publicado: (2021)

Changes in PCSK 9 and apolipoprotein B100 in Niemann–Pick disease after enzyme replacement therapy with olipudase alfa
por: Garside, Bethanie, et al.
Publicado: (2021)

Reconstruction of the Cytokine Signaling in Lysosomal Storage Diseases by Literature Mining and Network Analysis
por: Parolo, Silvia, et al.
Publicado: (2021)

Systems Pharmacology Model of Gastrointestinal Damage Predicts Species Differences and Optimizes Clinical Dosing Schedules
por: Shankaran, Harish, et al.
Publicado: (2017)

Quantitative system pharmacology as a legitimate approach to examine extrapolation strategies used to support pediatric drug development
por: Azer, Karim, et al.
Publicado: (2022)

Identification of a Novel Acid Sphingomyelinase Activity Associated with Recombinant Human Acid Ceramidase
por: He, Xingxuan, et al.
Publicado: (2023)

Pharmacological Inhibition of Acid Sphingomyelinase Prevents Uptake of SARS-CoV-2 by Epithelial Cells
por: Carpinteiro, Alexander, et al.
Publicado: (2020)

Recombinant Human Acid Sphingomyelinase as an Adjuvant to Sorafenib Treatment of Experimental Liver Cancer
por: Savić, Radoslav, et al.
Publicado: (2013)

QSPcc reduces bottlenecks in computational model simulations
por: Tomasoni, Danilo, et al.
Publicado: (2021)

Pharmacological Differentiation of Thrombomodulin Alfa and Activated Protein C on Coagulation and Fibrinolysis In Vitro
por: Tanaka, Kosuke, et al.
Publicado: (2018)

Modulation of Dietary Choline Uptake in a Mouse Model of Acid Sphingomyelinase Deficiency
por: Gaudioso, Ángel, et al.
Publicado: (2023)

Sphingomyelin 16:0 is a therapeutic target for neuronal death in acid sphingomyelinase deficiency
por: Gaudioso, Ángel, et al.
Publicado: (2023)

Linear-In-Flux-Expressions Methodology: Toward a Robust Mathematical Framework for Quantitative Systems Pharmacology Simulators
por: McQuade, Sean T, et al.
Publicado: (2017)

Recommendations for clinical monitoring of patients with acid sphingomyelinase deficiency (ASMD)
por: Wasserstein, Melissa, et al.
Publicado: (2018)

Consensus recommendation for a diagnostic guideline for acid sphingomyelinase deficiency
por: McGovern, Margaret M., et al.
Publicado: (2017)

Exocytosis of acid sphingomyelinase by wounded cells promotes endocytosis and plasma membrane repair
por: Tam, Christina, et al.
Publicado: (2010)

Acidic Sphingomyelinase (ASM) Is Necessary for Fas-induced GD3 Ganglioside Accumulation and Efficient Apoptosis of Lymphoid Cells
por: De Maria, Ruggero, et al.
Publicado: (1998)

Acid sphingomyelinase activity triggers microparticle release from glial cells
por: Bianco, Fabio, et al.
Publicado: (2009)

Acid sphingomyelinase modulates the autophagic process by controlling lysosomal biogenesis in Alzheimer’s disease
por: Lee, Jong Kil, et al.
Publicado: (2014)

Inhibition of fatty acid amide hydrolase prevents pathology in neurovisceral acid sphingomyelinase deficiency by rescuing defective endocannabinoid signaling
por: Bartoll, Adrián, et al.
Publicado: (2020)

Acid Sphingomyelinase Mediates Oxidized-LDL Induced Apoptosis in Macrophage via Endoplasmic Reticulum Stress
por: Zhao, Min, et al.
Publicado: (2016)

Sphingomyelinases and Liver Diseases
por: Insausti-Urkia, Naroa, et al.
Publicado: (2020)

The grand challenges in obstetric and pediatric pharmacology
por: Zajicek, Anne, et al.
Publicado: (2013)

A Quantitative Systems Pharmacology Platform to Investigate the Impact of Alirocumab and Cholesterol-Lowering Therapies on Lipid Profiles and Plaque Characteristics
por: Ming, Jeffrey E, et al.
Publicado: (2017)

Editorial: Insights in obstetric and pediatric pharmacology: 2021
por: Barrett, Jeffrey S.
Publicado: (2022)

Neutral sphingomyelinase-2, acid sphingomyelinase, and ceramide levels in COPD patients compared to controls
por: Lea, Simon R, et al.
Publicado: (2016)

Rational Pharmacological Approaches for Cognitive Dysfunction and Depression in Parkinson’s Disease
por: Sandoval-Rincón, Maritza, et al.
Publicado: (2015)

Cannot write session to /tmp/vufind_sessions/sess_oei7piqcn2pdsl8vpdu4j041p2