Genethon Presents Two Year Consolidated Results of Its Gene Therapy Trial for Duchenne Muscular Dystrophy: Maintenance of Motor Functions and Significant, Sustained Reduction in CPK Levels in Patients Treated at the Effective Dose at ASGCT 2025
GNT0004 product confirms its clinical efficacy with stabilization of motor functions in patients treated at the effective dose for up to 2 years. Sustained significant reduction in levels of creatine phosphokinase (CPK), a biomarker of muscle damage, with an average decrease of over 75% at 18 months in the 3 patients treated at the effective … [Read more…]