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2004 1
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38 results

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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.
Risdiplam for Prenatal Therapy of Spinal Muscular Atrophy.
Finkel RS, Hughes SH, Parker J, Civitello M, Lavado A, Mefford HC, Mueller L, Kletzl H; Prenatal SMA Risdiplam Study Group. Finkel RS, et al. Among authors: civitello m. N Engl J Med. 2025 Mar 13;392(11):1138-1140. doi: 10.1056/NEJMc2300802. Epub 2025 Feb 19. N Engl J Med. 2025. PMID: 39970420 No abstract available.
Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy.
Mercuri E, Darras BT, Chiriboga CA, Day JW, Campbell C, Connolly AM, Iannaccone ST, Kirschner J, Kuntz NL, Saito K, Shieh PB, Tulinius M, Mazzone ES, Montes J, Bishop KM, Yang Q, Foster R, Gheuens S, Bennett CF, Farwell W, Schneider E, De Vivo DC, Finkel RS; CHERISH Study Group. Mercuri E, et al. N Engl J Med. 2018 Feb 15;378(7):625-635. doi: 10.1056/NEJMoa1710504. N Engl J Med. 2018. PMID: 29443664 Free article. 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.
Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial.
McDonald CM, Marbán E, Hendrix S, Hogan N, Ruckdeschel Smith R, Eagle M, Finkel RS, Tian C, Janas J, Harmelink MM, Varadhachary AS, Taylor MD, Hor KN, Mayer OH, Henricson EK, Furlong P, Ascheim DD, Rogy S, Williams P, Marbán L; HOPE-2 Study Group. McDonald CM, et al. Lancet. 2022 Mar 12;399(10329):1049-1058. doi: 10.1016/S0140-6736(22)00012-5. Lancet. 2022. PMID: 35279258 Free article. Clinical Trial.
Long-term natural history in type II and III spinal muscular atrophy: a 4-year international study on the Hammersmith Functional Motor Scale Expanded.
Coratti G, Bovis F, Pera MC, Civitello M, Rohwer A, Salmin F, Glanzman AM, Montes J, Pasternak A, De Sanctis R, Dunaway Young S, Duong T, Mizzoni I, Milev E, Sframeli M, Morando S, Albamonte E, D'Amico A, Catteruccia M, Brolatti N, Pane M, Scoto M, Messina S, Escudero JE, De Waele L, Hirano M, Zolkipli-Cunningham Z, Darras BT, Bertini E, Nascimiento Osorio A, Bruno C, Goemans N, Sansone VA, Day J, Baranello G, Muntoni F, Finkel R, Mercuri E; ISMAC/international SMA consortium. Coratti G, et al. Among authors: civitello m. Eur J Neurol. 2024 Dec;31(12):e16517. doi: 10.1111/ene.16517. Epub 2024 Oct 11. Eur J Neurol. 2024. PMID: 39392101 Free PMC article.
Risdiplam in Presymptomatic Spinal Muscular Atrophy.
Finkel RS, Servais L, Vlodavets D, Zanoteli E, Mazurkiewicz-Bełdzińska M, Jong YJ, Navas-Nazario A, Al-Muhaizea M, Araujo APQC, Nelson L, Wang Y, Jaber B, Gorni K, Kletzl H, Palfreeman L, Rabbia M, Summers D, Gaki E, Wagner KR, Fontoura P, Farrar MA, Bertini E; RAINBOWFISH Study Group. Finkel RS, et al. N Engl J Med. 2025 Aug 14;393(7):671-682. doi: 10.1056/NEJMoa2410120. N Engl J Med. 2025. PMID: 40802943 Clinical Trial.
Upper limb function changes over 12 months in untreated SMA II and III individuals: an item-level analysis using the Revised Upper Limb Module.
Coratti G, Civitello M, Rohwer A, Albamonte E, Montes J, Glanzman AM, Pasternak A, De Sanctis R, Young SD, Duong T, Mizzoni I, Milev E, Sframeli M, Morando S, D'Amico A, Catteruccia M, Brolatti N, Pane M, Scoto M, Messina S, Hirano M, Zolkipli-Cunningham Z, Darras BT, Bertini E, Bruno C, Sansone VA, Salmin F, Day J, Baranello G, Pera MC, Muntoni F, Finkel RS, Mercuri E; International SMA Consortium (iSMAc). Coratti G, et al. Among authors: civitello m. Neuromuscul Disord. 2024 Nov;44:104449. doi: 10.1016/j.nmd.2024.08.006. Epub 2024 Aug 28. Neuromuscul Disord. 2024. PMID: 39299818 Free article.
Changes in abilities over the initial 12 months of nusinersen treatment for type II SMA.
Coratti G, Civitello M, Rohwer A, Salmin F, Glanzman AM, Montes J, Pasternak A, De Sanctis R, Young SD, Duong T, Mizzoni I, Milev E, Sframeli M, Morando S, Albamonte E, D'Amico A, Brolatti N, Pane M, Scoto M, Messina S, Hirano M, Zolkipli-Cunningham Z, Darras BT, Bertini E, Bruno C, Sansone VA, Day J, Baranello G, Pera MC, Muntoni F, Finkel R, Mercuri E. Coratti G, et al. Among authors: civitello m. Neuromuscul Disord. 2024 Aug;41:42-50. doi: 10.1016/j.nmd.2024.05.003. Epub 2024 May 9. Neuromuscul Disord. 2024. PMID: 38936290
38 results