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Page 1
Patients' Perceptions of Nusinersen Effects According to Their Responder Status.
Lilien C, Vrscaj E, Thapaliya G, Deconinck N, De Waele L, Duong T, Haberlová J, Kumhera M, Peirens G, Szabo L, Tahon V, Tang WJ, Benmhammed N, Médard L, Servais L. Lilien C, et al. Among authors: de waele l. J Clin Med. 2024 Jun 11;13(12):3418. doi: 10.3390/jcm13123418. J Clin Med. 2024. PMID: 38929947 Free PMC article.
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, de groot ijm. 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 Jul;31(7):e16282. doi: 10.1111/ene.16282. Epub 2024 Mar 20. Eur J Neurol. 2024. PMID: 38504654 Free PMC article.
105 results