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254 results

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Page 1
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: leone d. 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.
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. Among authors: leone d. EClinicalMedicine. 2023 May 5;59:101997. doi: 10.1016/j.eclinm.2023.101997. eCollection 2023 May. EClinicalMedicine. 2023. PMID: 37197706 Free PMC 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.
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: leone d. Eur J Pediatr. 2024 Apr 18. doi: 10.1007/s00431-024-05546-y. Online ahead of print. Eur J Pediatr. 2024. PMID: 38634892
Communicative development inventory in type 1 and presymptomatic infants with spinal muscular atrophy: a cohort study.
Buchignani B, Cicala G, Cumbo F, Ricci M, Capasso A, Ticci C, Mazzanti S, Brolatti N, Tosi M, Dosi C, Antonaci L, Coratti G, Pera MC, Leone D, Palermo C, Berti B, Frongia AL, Sacchini M, Bruno C, Masson R, D'Amico A, Battini R, Pane M, Mercuri E. Buchignani B, et al. Among authors: leone d. Arch Dis Child. 2024 Apr 18;109(5):395-401. doi: 10.1136/archdischild-2023-326613. Arch Dis Child. 2024. PMID: 38290776
Gain and loss of upper limb abilities in Duchenne muscular dystrophy patients: A 24-month study.
Coratti G, Pane M, Brogna C, D'Amico A, Pegoraro E, Bello L, Sansone VA, Albamonte E, Ferraroli E, Mazzone ES, Fanelli L, Messina S, Sframeli M, Catteruccia M, Cicala G, Capasso A, Ricci M, Frosini S, De Luca G, Rolle E, De Sanctis R, Forcina N, Norcia G, Passamano L, Scutifero M, Gardani A, Pini A, Monaco G, D'Angelo MG, Leone D, Zanin R, Vita GL, Panicucci C, Bruno C, Mongini T, Ricci F, Berardinelli A, Battini R, Masson R, Baranello G, Dosi C, Bertini E, Nigro V, Politano L, Mercuri E. Coratti G, et al. Among authors: leone d. Neuromuscul Disord. 2024 Jan;34:75-82. doi: 10.1016/j.nmd.2023.11.011. Epub 2023 Dec 3. Neuromuscul Disord. 2024. PMID: 38157655 Free article.
Longitudinal Analysis of PUL 2.0 Domains in Ambulant and Non-Ambulant Duchenne Muscular Dystrophy Patients: How do they Change in Relation to Functional Ability?
Pane M, Coratti G, Brogna C, Bovis F, D'Amico A, Pegoraro E, Bello L, Sansone V, Albamonte E, Ferraroli E, Mazzone ES, Fanelli L, Messina S, Catteruccia M, Cicala G, Ricci M, Frosini S, De Luca G, Rolle E, De Sanctis R, Forcina N, Norcia G, Passamano L, Gardani A, Pini A, Monaco G, D'Angelo MG, Capasso A, Leone D, Zanin R, Vita GL, Panicucci C, Bruno C, Mongini T, Ricci F, Berardinelli A, Battini R, Masson R, Baranello G, Dosi C, Bertini E, Politano L, Mercuri E. Pane M, et al. Among authors: leone d. J Neuromuscul Dis. 2023;10(4):567-574. doi: 10.3233/JND-221556. J Neuromuscul Dis. 2023. PMID: 37066919 Free PMC article.
254 results