Advancing rare disease therapeutics through digital twins: Opportunities in drug development and precision dosing.

ABSTRACT: Rare disease(s) (RD/RDs) are typically characterized by (i) genetically driven chronic, and life-threatening disease progression, (ii) delayed diagnoses, (iii) limited treatment options, and (iv) substantial economic burdens due to direct and indirect medical costs. Challenges in RD research include limited patient populations, sparse disease data, poorly understood pathophysiology and reduced trial funding for new exploratory therapies. In recent years, digital twin(s) (DT/DTs) are increasingly used for patient care, disease management, and resource optimization. They serve as virtual replicas of individual patients that enable simulation, prediction, and optimization of outcomes through real-time data integration and can facilitate advancements in treatment outcome and prediction of dise

SUBMITTER: Dette CMU 

PROVIDER: S-EPMC12703978 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature