Lessons Learned from Discontinued Clinical Developments in Duchenne Muscular Dystrophy

Lessons Learned from Discontinued Clinical Developments in Duchenne Muscular Dystrophy

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 approa...

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Bibliographic Details
Main Authors:	Theodora Markati, Liesbeth De Waele, Urlike Schara-Schmidt, Laurent Servais
Format:	Article
Language:	English
Published:	Frontiers Media S.A. 2021-11-01
Series:	Frontiers in Pharmacology
Subjects:	antisense oligonucleotides clinical trials duchenne muscular dystrophy dystrophin exon skipping randomized controlled clinical trials
Online Access:	https://www.frontiersin.org/articles/10.3389/fphar.2021.735912/full

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