Undisclosed Duchenne muscular dystrophy therapeutic / Novartis - LARVOL DELTA

Late Gadolinium Enhancement-Derived Heart Digital Twins Non-Invasively Predict Ventricular Tachycardia Risk in Duchenne Muscular Dystrophy (AHA 2024) - "LGE-derived digital twin models show strong potential for screening for VA risk in DMD patients. Future application of these models may help define optimal indications for ICD implantation in DMD."

Cardiomyopathy • Cardiovascular • Duchenne Muscular Dystrophy • Fibrosis • Genetic Disorders • Immunology • Muscular Dystrophy • Ventricular Tachycardia

A computational tool to optimize clinical trial parameter selection in Duchenne muscular dystrophy: A practical guide and case studies. (PubMed, CPT Pharmacometrics Syst Pharmacol) - "Two case studies, using input selection based on previous DMD clinical trials, provide realistic examples of how the tool can help optimize clinical trial design without the risk of decreasing statistical significance. This optimization allows researchers to mitigate the risk of designing trials that may be longer, larger, or more inclusive/exclusive than necessary."

Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Pediatrics