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Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial.
Dang UJ, Damsker JM, Guglieri M, Clemens PR, Perlman SJ, Smith EC, Horrocks I, Finkel RS, Mah JK, Deconinck N, Goemans NM, Haberlová 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. Dang UJ, et al. Among authors: de waele l. Neurology. 2024 Mar 12;102(5):e208112. doi: 10.1212/WNL.0000000000208112. Epub 2024 Feb 9. Neurology. 2024. PMID: 38335499 Free PMC article. Clinical Trial.
Lessons for future clinical trials in adults with Becker muscular dystrophy: Disease progression detected by muscle magnetic resonance imaging, clinical and patient-reported outcome measures.
De Wel B, Iterbeke L, Huysmans L, Peeters R, Goosens V, Dubuisson N, van den Bergh P, Van Parijs V, Remiche G, De Waele L, Maes F, Dupont P, Claeys KG. De Wel B, et al. Among authors: de waele l. Eur J Neurol. 2024 Mar 20:e16282. doi: 10.1111/ene.16282. Online ahead of print. Eur J Neurol. 2024. PMID: 38504654
Author Correction: Disrupted Ca2+ homeostasis and immunodeficiency in patients with functional IP3 receptor subtype 3 defects.
Neumann J, Van Nieuwenhove E, Terry LE, Staels F, Knebel TR, Welkenhuyzen K, Ahmadzadeh K, Baker MR, Gerbaux M, Willemsen M, Barber JS, Serysheva II, De Waele L, Vermeulen F, Schlenner S, Meyts I, Yule DI, Bultynck G, Schrijvers R, Humblet-Baron S, Liston A. Neumann J, et al. Among authors: de waele l. Cell Mol Immunol. 2023 Jan;20(1):114. doi: 10.1038/s41423-022-00960-4. Cell Mol Immunol. 2023. PMID: 36471115 Free PMC article. No abstract available.
Disrupted Ca2+ homeostasis and immunodeficiency in patients with functional IP3 receptor subtype 3 defects.
Neumann J, Van Nieuwenhove E, Terry LE, Staels F, Knebel TR, Welkenhuyzen K, Ahmadzadeh K, Baker MR, Gerbaux M, Willemsen M, Barber JS, Serysheva II, De Waele L, Vermeulen F, Schlenner S, Meyts I, Yule DI, Bultynck G, Schrijvers R, Humblet-Baron S, Liston A. Neumann J, et al. Among authors: de waele l. Cell Mol Immunol. 2023 Jan;20(1):11-25. doi: 10.1038/s41423-022-00928-4. Epub 2022 Oct 27. Cell Mol Immunol. 2023. PMID: 36302985 Free PMC article.
Gait classification for growing children with Duchenne muscular dystrophy.
Vandekerckhove I, Papageorgiou E, Hanssen B, De Beukelaer N, Van den Hauwe M, Goemans N, Van Campenhout A, De Waele L, De Groote F, Desloovere K. Vandekerckhove I, et al. Among authors: de waele l. Sci Rep. 2024 May 11;14(1):10828. doi: 10.1038/s41598-024-61231-y. Sci Rep. 2024. PMID: 38734731 Free PMC article.
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