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

• मुख्य लेखकों: Ruth Abrams (लेखक), Chanchala D. Kaddi (लेखक), Mengdi Tao (लेखक), Randolph J. Leiser (लेखक), Giulia Simoni (लेखक), Federico Reali (लेखक), John Tolsma (लेखक), Paul Jasper (लेखक), Zachary vanRijn (लेखक), Jing Li (लेखक), Bradley Niesner (लेखक), Jeffrey S. Barrett (लेखक), Luca Marchetti (लेखक), M. Judith Peterschmitt (लेखक), Karim Azer (लेखक), Susana Neves‐Zaph (लेखक)
• स्वरूप: पुस्तक
• प्रकाशित: Wiley, 2020-07-01T00:00:00Z.
• विषय: article
• ऑनलाइन पहुंच: 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...