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Page 1
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. 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.
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial.
Mercuri E, Muntoni F, Baranello G, Masson R, Boespflug-Tanguy O, Bruno C, Corti S, Daron A, Deconinck N, Servais L, Straub V, Ouyang H, Chand D, Tauscher-Wisniewski S, Mendonca N, Lavrov A; STR1VE-EU study group. Mercuri E, et al. Lancet Neurol. 2021 Oct;20(10):832-841. doi: 10.1016/S1474-4422(21)00251-9. Lancet Neurol. 2021. PMID: 34536405 Clinical Trial.
[No title available]
Montes J, Young SD, Mazzone E, Main M; International Spinal Muscular Atrophy Consortium Clinical Evaluator Working Group. Montes J, et al. Neuromuscul Disord. 2017 Jul 12:S0960-8966(17)30577-1. doi: 10.1016/j.nmd.2017.07.005. Online ahead of print. Neuromuscul Disord. 2017. PMID: 28917631 No abstract available.
Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study.
Chiriboga CA, Bruno C, Duong T, Fischer D, Mercuri E, Kirschner J, Kostera-Pruszczyk A, Jaber B, Gorni K, Kletzl H, Carruthers I, Martin C, Warren F, Scalco RS, Wagner KR, Muntoni F; JEWELFISH Study Group. Chiriboga CA, et al. Neurol Ther. 2023 Apr;12(2):543-557. doi: 10.1007/s40120-023-00444-1. Epub 2023 Feb 13. Neurol Ther. 2023. PMID: 36780114 Free PMC article.
Functional abilities, respiratory and cardiac function in a large cohort of adults with Duchenne muscular dystrophy treated with glucocorticoids.
Schiava M, Lofra RM, Bourke JP, Díaz-Manera J, James MK, Elseed MA, Malinova M, Michel-Sodhi J, Moat D, Ghimenton E, Mccallum M, Díaz CFB, Mayhew A, Wong K, Richardson M, Tasca G, Eglon G, Eagle M, Turner C, Heslop E, Straub V, Bettolo CM, Guglieri M. Schiava M, et al. Among authors: lofra rm. Eur J Neurol. 2024 Jun;31(6):e16267. doi: 10.1111/ene.16267. Epub 2024 Mar 31. Eur J Neurol. 2024. PMID: 38556893 Free PMC article.
Disease-associated comorbidities, medication records and anthropometric measures in adults with Duchenne muscular dystrophy.
Schiava M, Lofra RM, Bourke JP, James MK, Díaz-Manera J, Elseed MA, Michel-Sodhi J, Moat D, Mccallum M, Mayhew A, Ghimenton E, Díaz CFB, Malinova M, Wong K, Richardson M, Tasca G, Grover E, Robinson EJ, Tanner S, Eglon G, Behar L, Eagle M, Turner C, Verdú-Díaz J, Heslop E, Straub V, Bettolo CM, Guglieri M. Schiava M, et al. Among authors: lofra rm. Neuromuscul Disord. 2024 Aug;41:8-19. doi: 10.1016/j.nmd.2024.05.007. Epub 2024 May 14. Neuromuscul Disord. 2024. PMID: 38865917
Development of an International SMA Bulbar Assessment for Inter-professional Administration.
Dunaway Young S, McGrattan K, Johnson E, van der Heul M, Duong T, Bakke M, Werlauff U, Pasternak A, Cattaneo C, Hoffman K, Fanelli L, Breaks A, Allison K, Baranello G, Finkel R, Coratti G, Lofra RM. Dunaway Young S, et al. Among authors: lofra rm. J Neuromuscul Dis. 2023;10(4):639-652. doi: 10.3233/JND-221672. J Neuromuscul Dis. 2023. PMID: 37212069 Free PMC article.
Electrophysiologic features of SYT2 mutations causing a treatable neuromuscular syndrome.
Whittaker RG, Herrmann DN, Bansagi B, Hasan BA, Lofra RM, Logigian EL, Sowden JE, Almodovar JL, Littleton JT, Zuchner S, Horvath R, Lochmüller H. Whittaker RG, et al. Among authors: lofra rm. Neurology. 2015 Dec 1;85(22):1964-71. doi: 10.1212/WNL.0000000000002185. Epub 2015 Oct 30. Neurology. 2015. PMID: 26519543 Free PMC article.
Correction to: Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study.
Chiriboga CA, Bruno C, Duong T, Fischer D, Mercuri E, Kirschner J, Kostera-Pruszczyk A, Jaber B, Gorni K, Kletzl H, Carruthers I, Martin C, Warren F, Scalco RS, Wagner KR, Muntoni F; JEWELFISH Study Group. Chiriboga CA, et al. Neurol Ther. 2023 Oct;12(5):1799-1801. doi: 10.1007/s40120-023-00503-7. Neurol Ther. 2023. PMID: 37395990 Free PMC article. No abstract available.
14 results