Article
Quantitative Systems Pharmacology-Based Digital Twins Approach Supplements Clinical Trial Data for Enzyme Replacement Therapies in Pompe Disease.
Clinical pharmacology and therapeutics - 1 Feb 2025
Kaddi Chanchala, Tao Mengdi, Bergeler Silke, George Kelly, Geerts Hugo, van der Graaf Piet H, Batista Julie L, Foster Meredith, Ortemann-Renon Catherine, Zaher Atef, An Haack Kristina, Zaph Susana
Abstract excerpt
Pompe disease is a rare, progressive neuromuscular disease caused by deficient lysosomal glycogen degradation, and includes both late-onset (LOPD) and severe infantile-onset (IOPD) phenotypes. Due to very small patient numbers in IOPD and the high phenotypic heterogeneity observed in this population, a quantitative systems pharmacology (QSP)-based "digital twin" approach was developed to perform an in silico...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.