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Spinal muscular atrophy care in the COVID-19 pandemic era.
Veerapandiyan A, Connolly AM, Finkel RS, Arya K, Mathews KD, Smith EC, Castro D, Butterfield RJ, Parsons JA, Servais L, Kuntz N, Rao VK, Brandsema JF, Mercuri E, Ciafaloni E. Veerapandiyan A, et al. Among authors: servais l. Muscle Nerve. 2020 Jul;62(1):46-49. doi: 10.1002/mus.26903. Epub 2020 May 3. Muscle Nerve. 2020. PMID: 32329921 Free PMC article. Review.
International workshop on assessment of upper limb function in Duchenne Muscular Dystrophy: Rome, 15-16 February 2012.
Mercuri E, McDonald C, Mayhew A, Florence J, Mazzone E, Bianco F, Decostre V, Servais L, Ricotti V, Goemans N, Vroom E. Mercuri E, et al. Among authors: servais l. Neuromuscul Disord. 2012 Nov;22(11):1025-8. doi: 10.1016/j.nmd.2012.06.006. Epub 2012 Jul 13. Neuromuscul Disord. 2012. PMID: 22795657 Free PMC article. No abstract available.
A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy.
Victor RG, Sweeney HL, Finkel R, McDonald CM, Byrne B, Eagle M, Goemans N, Vandenborne K, Dubrovsky AL, Topaloglu H, Miceli MC, Furlong P, Landry J, Elashoff R, Cox D; Tadalafil DMD Study Group. Victor RG, et al. Neurology. 2017 Oct 24;89(17):1811-1820. doi: 10.1212/WNL.0000000000004570. Epub 2017 Sep 29. Neurology. 2017. PMID: 28972192 Free PMC article. Clinical Trial.
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.
Finkel RS, Mercuri E, Darras BT, Connolly AM, Kuntz NL, Kirschner J, Chiriboga CA, Saito K, Servais L, Tizzano E, Topaloglu H, Tulinius M, Montes J, Glanzman AM, Bishop K, Zhong ZJ, Gheuens S, Bennett CF, Schneider E, Farwell W, De Vivo DC; ENDEAR Study Group. Finkel RS, et al. Among authors: servais l. N Engl J Med. 2017 Nov 2;377(18):1723-1732. doi: 10.1056/NEJMoa1702752. N Engl J Med. 2017. PMID: 29091570 Free article. Clinical Trial.
Long-term data with idebenone on respiratory function outcomes in patients with Duchenne muscular dystrophy.
Servais L, Straathof CSM, Schara U, Klein A, Leinonen M, Hasham S, Meier T, De Waele L, Gordish-Dressman H, McDonald CM, Mayer OH, Voit T, Mercuri E, Buyse GM; SYROS and CINRG DNHS Investigators. Servais L, et al. Neuromuscul Disord. 2020 Jan;30(1):5-16. doi: 10.1016/j.nmd.2019.10.008. Epub 2019 Nov 5. Neuromuscul Disord. 2020. PMID: 31813614 Free article. Clinical Trial.
RESTORE: A Prospective Multinational Registry of Patients with Genetically Confirmed Spinal Muscular Atrophy - Rationale and Study Design.
Finkel RS, Day JW, De Vivo DC, Kirschner J, Mercuri E, Muntoni F, Shieh PB, Tizzano E, Desguerre I, Quijano-Roy S, Saito K, Droege M, Dabbous O, Khan F, Renault L, Anderson FA, Servais L. Finkel RS, et al. Among authors: servais l. J Neuromuscul Dis. 2020;7(2):145-152. doi: 10.3233/JND-190451. J Neuromuscul Dis. 2020. PMID: 32039859 Free PMC article.
Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy.
Frank DE, Schnell FJ, Akana C, El-Husayni SH, Desjardins CA, Morgan J, Charleston JS, Sardone V, Domingos J, Dickson G, Straub V, Guglieri M, Mercuri E, Servais L, Muntoni F; SKIP-NMD Study Group. Frank DE, et al. Among authors: servais l. Neurology. 2020 May 26;94(21):e2270-e2282. doi: 10.1212/WNL.0000000000009233. Epub 2020 Mar 5. Neurology. 2020. PMID: 32139505 Free PMC article. Clinical Trial.
European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy.
Kirschner J, Butoianu N, Goemans N, Haberlova J, Kostera-Pruszczyk A, Mercuri E, van der Pol WL, Quijano-Roy S, Sejersen T, Tizzano EF, Ziegler A, Servais L, Muntoni F. Kirschner J, et al. Among authors: servais l. Eur J Paediatr Neurol. 2020 Sep;28:38-43. doi: 10.1016/j.ejpn.2020.07.001. Epub 2020 Jul 9. Eur J Paediatr Neurol. 2020. PMID: 32763124 Free PMC article.
255 results