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Page 1
Treatment strategies for patients with spinal muscular atrophy.
De Waele L, Servais L. De Waele L, et al. Among authors: servais l. Expert Rev Neurother. 2024 Dec 10:1-3. doi: 10.1080/14737175.2024.2439486. Online ahead of print. Expert Rev Neurother. 2024. PMID: 39648756 Free article. No abstract available.
Natural history of Type 2 and 3 spinal muscular atrophy: 2-year NatHis-SMA study.
Annoussamy M, Seferian AM, Daron A, Péréon Y, Cances C, Vuillerot C, De Waele L, Laugel V, Schara U, Gidaro T, Lilien C, Hogrel JY, Carlier P, Fournier E, Lowes L, Gorni K, Ly-Le Moal M, Hellbach N, Seabrook T, Czech C, Hermosilla R, Servais L; NatHis-SMA study group. Annoussamy M, et al. Among authors: servais l. Ann Clin Transl Neurol. 2021 Feb;8(2):359-373. doi: 10.1002/acn3.51281. Epub 2020 Dec 24. Ann Clin Transl Neurol. 2021. PMID: 33369268 Free PMC article.
Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial.
Mercuri E, Deconinck N, Mazzone ES, Nascimento A, Oskoui M, Saito K, Vuillerot C, Baranello G, Boespflug-Tanguy O, Goemans N, Kirschner J, Kostera-Pruszczyk A, Servais L, Gerber M, Gorni K, Khwaja O, Kletzl H, Scalco RS, Staunton H, Yeung WY, Martin C, Fontoura P, Day JW; SUNFISH Study Group. Mercuri E, et al. Among authors: servais l. Lancet Neurol. 2022 Jan;21(1):42-52. doi: 10.1016/S1474-4422(21)00367-7. Lancet Neurol. 2022. PMID: 34942136
Onasemnogene abeparvovec for the treatment of spinal muscular atrophy.
McMillan HJ, Proud CM, Farrar MA, Alexander IE, Muntoni F, Servais L. McMillan HJ, et al. Among authors: servais l. Expert Opin Biol Ther. 2022 Sep;22(9):1075-1090. doi: 10.1080/14712598.2022.2066471. Epub 2022 May 2. Expert Opin Biol Ther. 2022. PMID: 35437095 Free article.
2024 update: European consensus statement on gene therapy for spinal muscular atrophy.
Kirschner J, Bernert G, Butoianu N, De Waele L, Fattal-Valevski A, Haberlova J, Moreno T, Klein A, Kostera-Pruszczyk A, Mercuri E, Quijano-Roy S, Sejersen T, Tizzano EF, van der Pol WL, Wallace S, Zafeiriou D, Ziegler A, Muntoni F, Servais L. Kirschner J, et al. Among authors: servais l. Eur J Paediatr Neurol. 2024 Jul;51:73-78. doi: 10.1016/j.ejpn.2024.06.001. Epub 2024 Jun 8. Eur J Paediatr Neurol. 2024. PMID: 38878702 Free article.
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: servais l. J Clin Med. 2024 Jun 11;13(12):3418. doi: 10.3390/jcm13123418. J Clin Med. 2024. PMID: 38929947 Free PMC article.
Risdiplam in Type 1 Spinal Muscular Atrophy.
Baranello G, Darras BT, Day JW, Deconinck N, Klein A, Masson R, Mercuri E, Rose K, El-Khairi M, Gerber M, Gorni K, Khwaja O, Kletzl H, Scalco RS, Seabrook T, Fontoura P, Servais L; FIREFISH Working Group. Baranello G, et al. Among authors: servais l. N Engl J Med. 2021 Mar 11;384(10):915-923. doi: 10.1056/NEJMoa2009965. Epub 2021 Feb 24. N Engl J Med. 2021. PMID: 33626251 Clinical Trial.
281 results