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Year Number of Results
2011 2
2012 5
2013 5
2014 5
2015 6
2016 8
2017 5
2018 5
2019 3
2020 14
2021 13
2022 12
2023 15
2024 7

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94 results

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Page 1
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. Lancet Neurol. 2022 Jan;21(1):42-52. doi: 10.1016/S1474-4422(21)00367-7. Lancet Neurol. 2022. PMID: 34942136
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. 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.
Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial.
Servais L, Mercuri E, Straub V, Guglieri M, Seferian AM, Scoto M, Leone D, Koenig E, Khan N, Dugar A, Wang X, Han B, Wang D, Muntoni F; SKIP-NMD Study Group. Servais L, et al. Among authors: seferian am. Nucleic Acid Ther. 2022 Feb;32(1):29-39. doi: 10.1089/nat.2021.0043. Epub 2021 Nov 17. Nucleic Acid Ther. 2022. PMID: 34788571 Free PMC article. Clinical Trial.
Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls.
Darras BT, Masson R, Mazurkiewicz-Bełdzińska M, Rose K, Xiong H, Zanoteli E, Baranello G, Bruno C, Vlodavets D, Wang Y, El-Khairi M, Gerber M, Gorni K, Khwaja O, Kletzl H, Scalco RS, Fontoura P, Servais L; FIREFISH Working Group. Darras BT, et al. N Engl J Med. 2021 Jul 29;385(5):427-435. doi: 10.1056/NEJMoa2102047. N Engl J Med. 2021. PMID: 34320287
Association between Initial Treatment Strategy and Long-Term Survival in Pulmonary Arterial Hypertension.
Boucly A, Savale L, Jaïs X, Bauer F, Bergot E, Bertoletti L, Beurnier A, Bourdin A, Bouvaist H, Bulifon S, Chabanne C, Chaouat A, Cottin V, Dauphin C, Degano B, De Groote P, Favrolt N, Feng Y, Horeau-Langlard D, Jevnikar M, Jutant EM, Liang Z, Magro P, Mauran P, Moceri P, Mornex JF, Palat S, Parent F, Picard F, Pichon J, Poubeau P, Prévot G, Renard S, Reynaud-Gaubert M, Riou M, Roblot P, Sanchez O, Seferian A, Tromeur C, Weatherald J, Simonneau G, Montani D, Humbert M, Sitbon O. Boucly A, et al. Among authors: seferian a. Am J Respir Crit Care Med. 2021 Oct 1;204(7):842-854. doi: 10.1164/rccm.202009-3698OC. Am J Respir Crit Care Med. 2021. PMID: 34185620 Free 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. 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.
Sarcoidosis-Associated Pulmonary Hypertension.
Savale L, Dorfmüller P, Boucly A, Jaïs X, Lacoste-Palasset T, Jevnikar M, Seferian A, Humbert M, Sitbon O, Montani D. Savale L, et al. Among authors: seferian a. Clin Chest Med. 2024 Mar;45(1):185-197. doi: 10.1016/j.ccm.2023.08.007. Epub 2023 Sep 1. Clin Chest Med. 2024. PMID: 38245366 Review.
Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial.
Masson R, Mazurkiewicz-Bełdzińska M, Rose K, Servais L, Xiong H, Zanoteli E, Baranello G, Bruno C, Day JW, Deconinck N, Klein A, Mercuri E, Vlodavets D, Wang Y, Dodman A, El-Khairi M, Gorni K, Jaber B, Kletzl H, Gaki E, Fontoura P, Darras BT; FIREFISH Study Group. Masson R, et al. Lancet Neurol. 2022 Dec;21(12):1110-1119. doi: 10.1016/S1474-4422(22)00339-8. Epub 2022 Oct 14. Lancet Neurol. 2022. PMID: 36244364 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.
Drug-induced pulmonary arterial hypertension: a recent outbreak.
Montani D, Seferian A, Savale L, Simonneau G, Humbert M. Montani D, et al. Among authors: seferian a. Eur Respir Rev. 2013 Sep 1;22(129):244-50. doi: 10.1183/09059180.00003313. Eur Respir Rev. 2013. PMID: 23997051 Free PMC article. Review.
94 results