Quantitative magnetic resonance imaging measures as biomarkers of disease progression in boys with Duchenne muscular dystrophy: a phase 2 trial of domagrozumab

Duchenne muscular dystrophy (DMD) is a progressive, neuromuscular disorder caused by mutations in the DMD gene that results in a lack of functional dystrophin protein. Herein, we report the use of quantitative magnetic resonance imaging (MRI) measures as biomarkers in the context of a multicenter ph...

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Detalles Bibliográficos
Autores principales: Sherlock, Sarah P., Palmer, Jeffrey, Wagner, Kathryn R., Abdel-Hamid, Hoda Z., Bertini, Enrico, Tian, Cuixia, Mah, Jean K., Kostera-Pruszczyk, Anna, Muntoni, Francesco, Guglieri, Michela, Brandsema, John F., Mercuri, Eugenio, Butterfield, Russell J., McDonald, Craig M., Charnas, Lawrence, Marraffino, Shannon
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Berlin Heidelberg 2022
Materias:
Original Communication
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9294028/
https://www.ncbi.nlm.nih.gov/pubmed/35396602
http://dx.doi.org/10.1007/s00415-022-11084-0

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