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Multi-omics profiling of CSF from spinal muscular atrophy type 3 patients after nusinersen treatment: a 2-year follow-up multicenter retrospective study.
Faravelli I, Gagliardi D, Abati E, Meneri M, Ongaro J, Magri F, Parente V, Petrozzi L, Ricci G, Farè F, Garrone G, Fontana M, Caruso D, Siciliano G, Comi GP, Govoni A, Corti S, Ottoboni L. Faravelli I, et al. Cell Mol Life Sci. 2023 Aug 5;80(8):241. doi: 10.1007/s00018-023-04885-7. Cell Mol Life Sci. 2023. PMID: 37543540 Free PMC article.
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. Eur J Neurol. 2024 Aug;31(8):e16309. doi: 10.1111/ene.16309. Epub 2024 Apr 24. Eur J Neurol. 2024. PMID: 38656662 Free PMC article.
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. 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.
Prognostic factors for tube feeding in type I SMA patients treated with disease-modifying therapies: a cohort study.
Pane M, Stanca G, Coratti G, D' Amico A, Sansone VA, Berti B, Fanelli L, Albamonte E, Ausili Cefaro C, Cerchiari A, Catteruccia M, De Sanctis R, Leone D, Palermo C, Buchignani B, Onesimo R, Kuczynska EM, Tosi M, Pera MC, Bravetti C, Tiziano FD, Bertini E, Mercuri E. Pane M, et al. Eur J Pediatr. 2024 Nov;183(11):4735-4745. doi: 10.1007/s00431-024-05735-9. Epub 2024 Aug 29. Eur J Pediatr. 2024. PMID: 39210071 Free PMC article.