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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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Detalles Bibliográficos
Autores principales: Markati, Theodora, De Waele, Liesbeth, Schara-Schmidt, Urlike, Servais, Laurent
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8591262/
https://www.ncbi.nlm.nih.gov/pubmed/34790118
http://dx.doi.org/10.3389/fphar.2021.735912