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Page 1
Type I spinal muscular atrophy and disease modifying treatments: a nationwide study in children born since 2016.
Pera MC, Coratti G, Pane M, Masson R, Sansone VA, D'Amico A, Catteruccia M, Agosto C, Varone A, Bruno C, Messina S, Ricci F, Bruno I, Procopio E, Pini A, Siliquini S, Zanin R, Albamonte E, Berardinelli A, Mastella C, Baranello G, Previtali SC, Trabacca A, Bravetti C, Gagliardi D, Filosto M, de Sanctis R, Finkel R, Mercuri E; Italian SMA type I Study Group. Pera MC, et al. EClinicalMedicine. 2024 Nov 30;78:102967. doi: 10.1016/j.eclinm.2024.102967. eCollection 2024 Dec. EClinicalMedicine. 2024. PMID: 39687428 Free PMC article.
Prevalence of Duchenne muscular dystrophy in Italy: a nationwide survey.
Capasso A, Cicala G, Ricci M, Pane M, D'Amico A, Bruno C, Sansone VA, Messina S, Bello L, Pegoraro E, D'Angelo MG, Masson R, Berardinelli A, Pini A, Ricci F, Mongini TE, Coccia M, Nigro V, Trabacca A, Filosto M, Comi G, Magri F, Barp A, Battini R, Previtali SC, Valentino ML, Diella E, Dosi C, Ruggiero L, Siciliano G, Ricci G, Catteruccia M, Arpaia C, Coratti G, Norcia G, Bonanno S, Verriello L, Agosto C, Varone A, Ferlini A, Maioli MA, Brogna C, Siliquini S, Bruno I, Panicucci C, Allegra C, Albamonte E, Mercuri E; Italian DMD group. Capasso A, et al. Eur J Pediatr. 2024 Dec 16;184(1):86. doi: 10.1007/s00431-024-05903-x. Eur J Pediatr. 2024. PMID: 39680193
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. Among authors: stanca g. 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.
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.
Early neurological signs in infants identified through neonatal screening for SMA: do they predict outcome?
Pane M, Stanca G, Ticci C, Cutrona C, De Sanctis R, Pirinu M, Coratti G, Palermo C, Berti B, Leone D, Sacchini M, Cerboneschi M, Fanelli L, Norcia G, Forcina N, Capasso A, Cicala G, Antonaci L, Ricci M, Pera MC, Bravetti C, Donati MA, Procopio E, Abiusi E, Vaisfeld A, Onesimo R, Tiziano FD, Mercuri E. Pane M, et al. Among authors: stanca g. Eur J Pediatr. 2024 Jul;183(7):2995-2999. doi: 10.1007/s00431-024-05546-y. Epub 2024 Apr 18. Eur J Pediatr. 2024. PMID: 38634892 Free PMC article.
Can the CHOP-INTEND be used as An Outcome Measure in the First Months of Age? Implications for Clinical Trials and Real World Data.
Cutrona C, de Sanctis R, Coratti G, Capasso A, Ricci M, Stanca G, Carnicella S, Utlulig M, Bersani G, Lazzareschi I, Leoni C, Buonsenso D, Luciano R, Vento G, Finkel RS, Pane M, Mercuri E. Cutrona C, et al. Among authors: stanca g. J Neuromuscul Dis. 2024;11(1):85-90. doi: 10.3233/JND-221644. J Neuromuscul Dis. 2024. PMID: 37980678 Free PMC article.
Patient reported outcome measure for upper limb in Duchenne muscular dystrophy: correlation with PUL2.0.
Cicala G, Pane M, Coratti G, Brogna C, Fanelli L, Norcia G, Forcina N, Mazzone E, Stanca G, Ferrante R, Vento A, Ferraroli E, Ricci M, Capasso A, Leone D, Palermo C, Berti B, Cutrona C, Mahyew A, Duong T, Goemans N, Vroom E, Mercuri E. Cicala G, et al. Among authors: stanca g. Neuromuscul Disord. 2023 Sep;33(9):69-73. doi: 10.1016/j.nmd.2023.07.003. Epub 2023 Jul 22. Neuromuscul Disord. 2023. PMID: 37612177 Free article.
Assessing Prevalence and Characteristics of Oro-bulbar Involvement in Children and Adults with SMA Type 2 and 3 Using a Multimodal Approach.
Trucco F, Salmin F, Lizio A, Coratti G, Albamonte E, Frisoni MC, Mauro L, Carraro E, Palazzo G, Lops J, Cattaneo C, Pozzi S, Casiraghi J, Di Bari A, Berti B, Stanca G, Ricci M, Pane M, Heatwole C, Dilek N, Mercuri E, Sansone VA. Trucco F, et al. Among authors: stanca g. Dysphagia. 2023 Dec;38(6):1568-1580. doi: 10.1007/s00455-023-10584-z. Epub 2023 Jun 8. Dysphagia. 2023. PMID: 37289231 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.
11 results