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Article

Lessons Learned from Discontinued Clinical Developments in Duchenne Muscular Dystrophy

1 Nov 2021

Abstract excerpt

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...

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PMID: 34790118
DOI: 10.3389/fphar.2021.735912

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