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Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy; A Randomized Controlled Trial

Lookup NU author(s): Professor Michela GuglieriORCiD, Dr Jana Haberlova, Professor Volker StraubORCiD, Dr Liesbeth De Waele

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Author(s): Dang UJ, Damsker J, Guglieri M, Clemens PR, Perlman SJ, Smith EC, Horrocks I, Finkel RS, Mah JK, Deconinck N, Goemans NM, Haberlova J, Straub V, Mengle-Gaw L, Schwartz BD, Harper A, Shieh PB, De Waele L, Castro D, Yang ML, Ryan MM, McDonald CM, Tulinius M, Webster RI, Mcmillan HJ, Kuntz N, Rao VK, Baranello G, Spinty S, Childs AM, Sbrocchi AM, Selby KA, Monduy M, Nevo Y, Vilchez JJ, Nascimento-Osorio A, Niks EH, De Groot IJM, Katsalouli M, Van Den Anker JN, Ward LM, Leinonen M, D'Alessandro AL, Hoffman EP

Publication type: Article

Publication status: Published

Journal: Neurology

Year: 2024

Volume: 102

Issue: 5

Print publication date: 12/03/2024

Online publication date: 09/02/2024

Acceptance date: 23/12/2023

Date deposited: 19/03/2024

ISSN (print): 0028-3878

ISSN (electronic): 1526-632X

Publisher: Wolters Kluwer Health

URL: https://doi.org/10.1212/WNL.0000000000208112

DOI: 10.1212/WNL.0000000000208112

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