A Quantitative Systems Pharmacology Model of Gaucher Disease Type 1 Provides Mechanistic Insight Into the Response to Substrate Reduction Therapy With Eliglustat

• Main Authors: Ruth Abrams (Author), Chanchala D. Kaddi (Author), Mengdi Tao (Author), Randolph J. Leiser (Author), Giulia Simoni (Author), Federico Reali (Author), John Tolsma (Author), Paul Jasper (Author), Zachary vanRijn (Author), Jing Li (Author), Bradley Niesner (Author), Jeffrey S. Barrett (Author), Luca Marchetti (Author), M. Judith Peterschmitt (Author), Karim Azer (Author), Susana Neves‐Zaph (Author)
• Format: Book
• Published: Wiley, 2020-07-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Gaucher's disease type 1 (GD1) leads to significant morbidity and mortality through clinical manifestations, such as splenomegaly, hematological complications, and bone disease. Two types of therapies are currently approved for GD1: enzyme replacement therapy (ERT), and substrate reduction ther...