Current Trends in Duchenne Muscular Dystrophy Research and Therapy: 3D Cardiac Modelling.

ABSTRACT: Duchenne muscular dystrophy (DMD), caused by dystrophin deficiency, presents a multifaceted challenge that affects both skeletal muscle function and cardiomyocyte homeostasis, causing progressive degeneration and life-threatening cardiac complications by adolescence. Current treatments fail to prevent poor prognoses, and while FDA-approved therapies show promise in targeting dystrophin restoration, including RNA-based approaches and microdystrophin gene therapy, clinical evidence supporting their efficacy remains limited. Substantial challenges persist, particularly in achieving effective cardiac targeting, ensuring long-term safety and developing scalable treatments. Alternative therapies addressing muscle and cardiac pathophysiology are being explored alongside dystrophin-based approache

SUBMITTER: Przymuszala M 

PROVIDER: S-EPMC12776602 | biostudies-literature | 2026 Feb

REPOSITORIES: biostudies-literature