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Page 1
Determining minimal clinically important differences in the Hammersmith Functional Motor Scale Expanded for untreated spinal muscular atrophy patients: An international study.
Coratti G, Bovis F, Pera MC, Scoto M, Montes J, Pasternak A, Mayhew A, Muni-Lofra R, Duong T, Rohwer A, Dunaway Young S, Civitello M, Salmin F, Mizzoni I, Morando S, Pane M, Albamonte E, D'Amico A, Brolatti N, Sframeli M, Marini-Bettolo C, Sansone VA, Bruno C, Messina S, Bertini E, Baranello G, Day J, Darras BT, De Vivo DC, Hirano M, Muntoni F, Finkel R, Mercuri E; ISMAC group. Coratti G, et al. Among authors: morando s. Eur J Neurol. 2024 Apr 24:e16309. doi: 10.1111/ene.16309. Online ahead of print. Eur J Neurol. 2024. PMID: 38656662
Sometimes they come back: New and old spinal muscular atrophy adults in the era of nusinersen.
Sansone VA, Coratti G, Pera MC, Pane M, Messina S, Salmin F, Albamonte E, De Sanctis R, Sframeli M, Di Bella V, Morando S, d'Amico A, Frongia AL, Antonaci L, Pirola A, Pedemonte M, Bertini E, Bruno C, Mercuri E; Italian ISMAC group. Sansone VA, et al. Among authors: morando s. Eur J Neurol. 2021 Feb;28(2):602-608. doi: 10.1111/ene.14567. Epub 2020 Oct 31. Eur J Neurol. 2021. PMID: 33012052 Free article.
Nusinersen in pediatric and adult patients with type III spinal muscular atrophy.
Pera MC, Coratti G, Bovis F, Pane M, Pasternak A, Montes J, Sansone VA, Dunaway Young S, Duong T, Messina S, Mizzoni I, D'Amico A, Civitello M, Glanzman AM, Bruno C, Salmin F, Morando S, De Sanctis R, Sframeli M, Antonaci L, Frongia AL, Rohwer A, Scoto M, De Vivo DC, Darras BT, Day J, Martens W, Patanella KA, Bertini E, Muntoni F, Finkel R, Mercuri E; iSMAC group. Pera MC, et al. Among authors: morando s. Ann Clin Transl Neurol. 2021 Aug;8(8):1622-1634. doi: 10.1002/acn3.51411. Epub 2021 Jun 24. Ann Clin Transl Neurol. 2021. PMID: 34165911 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. 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.
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.
Nusinersen efficacy data for 24-month in type 2 and 3 spinal muscular atrophy.
Pane M, Coratti G, Pera MC, Sansone VA, Messina S, d'Amico A, Bruno C, Salmin F, Albamonte E, De Sanctis R, Sframeli M, Di Bella V, Morando S, Palermo C, Frongia AL, Antonaci L, Capasso A, Catteruccia M, Longo A, Ricci M, Cutrona C, Pirola A, Bravetti C, Pedemonte M, Brolatti N, Bertini E, Mercuri E; Italian ISMAC group. Pane M, et al. Among authors: morando s. Ann Clin Transl Neurol. 2022 Mar;9(3):404-409. doi: 10.1002/acn3.51514. Epub 2022 Feb 15. Ann Clin Transl Neurol. 2022. PMID: 35166467 Free PMC article.
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.
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.
Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from other therapies.
Pane M, Berti B, Capasso A, Coratti G, Varone A, D'Amico A, Messina S, Masson R, Sansone VA, Donati MA, Agosto C, Bruno C, Ricci F, Pini A, Gagliardi D, Filosto M, Corti S, Leone D, Palermo C, Onesimo R, De Sanctis R, Ricci M, Bitetti I, Sframeli M, Dosi C, Albamonte E, Ticci C, Brolatti N, Bertini E, Finkel R, Mercuri E; ITASMAc group. Pane M, et al. EClinicalMedicine. 2023 May 5;59:101997. doi: 10.1016/j.eclinm.2023.101997. eCollection 2023 May. EClinicalMedicine. 2023. PMID: 37197706 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.
29 results