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pro vyhledávání: '"Roxana Dreghici"'
Autor:
Francesco Muntoni, Barry J. Byrne, Hugh J. McMillan, Monique M. Ryan, Brenda L. Wong, Juergen Dukart, Amita Bansal, Valerie Cosson, Roxana Dreghici, Maitea Guridi, Michael Rabbia, Hannah Staunton, Giridhar S. Tirucherai, Karl Yen, Xiling Yuan, Kathryn R. Wagner, the Taldefgrobep Alfa Study Group
Publikováno v:
Neurology and Therapy, Vol 13, Iss 1, Pp 183-219 (2024)
Abstract Introduction Duchenne muscular dystrophy (DMD) is a genetic muscle disorder that manifests during early childhood and is ultimately fatal. Recently approved treatments targeting the genetic cause of DMD are limited to specific subpopulations
Externí odkaz:
https://doaj.org/article/202c472942a04da4895185c648dc27dc