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

Internet

https://www.frontiersin.org/articles/10.3389/fphar.2021.735912/full

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