Lessons Learned from Discontinued Clinical Developments in Duchenne Muscular Dystrophy.

ABSTRACT: Duchenne muscular dystrophy (DMD) is an X-linked condition caused by a deficiency of functional dystrophin protein. Patients experience progressive muscle weakness, cardiomyopathy and have a decreased life expectancy. Standards of care, including treatment with steroids, and multidisciplinary approaches have extended the life expectancy and improved the quality of life of patients. In the last 30 years, several compounds have been assessed in preclinical and clinical studies for their ability to restore functional dystrophin levels or to modify pathways involved in DMD pathophysiology. However, there is still an unmet need with regards to a disease-modifying treatment for DMD and the attrition rate between early-phase and late-phase clinical development remains high. Currently, there are 4

SUBMITTER: Markati T 

PROVIDER: S-EPMC8591262 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature