Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy

Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy

Vamorolone is a dissociative agonist of the glucocorticoid receptor that has shown similar efficacy and reduced safety concerns in comparison with prednisone in Duchenne muscular dystrophy (DMD). This study was conducted to determine the efficacy and safety of vamorolone over 48 weeks and to study c...

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Ngā kaituhi matua: Utkarsh J. Dang, Jesse M. Damsker, Michela Guglieri, Paula R. Clemens, Seth J. Perlman, Edward C. Smith, Iain Horrocks, Richard S. Finkel, Jean K. Mah, Nicolas Deconinck, Nathalie Goemans, Jana Haberlová, Volker Straub, L. Mengle-Gaw, Benjamin D. Schwartz, Amy Harper, Perry B. Shieh, Liesbeth De Waele, Diana Castro, Michele Yang, Monique M. Ryan, Craig M. McDonald, M. Tulinius, Richard Webster, Hugh J. McMillan, Nancy L. Kuntz, Vamshi K. Rao, Giovanni Baranello, Stefan Spinty, Anne‐Marie Childs, Annie M. Sbrocchi, Kathryn Selby, Migvis Monduy, Yoram Nevo, Juan J. Vílchez, Andres Nascimento-Osorio, Erik H. Niks, Imelda J. M. de Groot, Marina Katsalouli, John N. van den Anker, Leanne M. Ward, Mika Leinonen, Andrea L. D'Alessandro, Eric P. Hoffman
Hōputu: Artigo
Reo:Ingarihi
I whakaputaina: 2024
Urunga tuihono:https://doi.org/10.1212/wnl.0000000000208112
https://www.neurology.org/doi/pdfdirect/10.1212/WNL.0000000000208112
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Ipurangi

https://doi.org/10.1212/wnl.0000000000208112
https://www.neurology.org/doi/pdfdirect/10.1212/WNL.0000000000208112

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