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266 results

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Page 1
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, d amico a. 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.
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.
Genetic risk factors for Mesoamerican nephropathy.
Friedman DJ, Leone DA, Amador JJ, Kupferman J, Francey LJ, Lopez-Pilarte D, Lau J, Delgado I, Yih WK, Salinas A, Wang M, Genovese G, Shah S, Kelly J, Tattersfield CF, Raines NH, Amador M, Dias L, Pitsillides A, Ramirez-Rubio O, Amador AG, Cortopassi M, Applebaum KM, Alper SL, Banks AS, McClean MD, Leibler JH, Scammell MK, Dupuis J, Brooks DR. Friedman DJ, et al. Among authors: leone da. Proc Natl Acad Sci U S A. 2024 Dec 3;121(49):e2404848121. doi: 10.1073/pnas.2404848121. Epub 2024 Nov 25. Proc Natl Acad Sci U S A. 2024. PMID: 39585978
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: leone d. 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.
Derangement in Nicotinamide Adenine Dinucleotide Metabolism is Observed During Acute Kidney Injury Among Male Agricultural Workers at Risk for Mesoamerican Nephropathy.
Raines NH, Leone DA, Amador JJ, Lopez-Pilarte D, Ramírez-Rubio O, Delgado IS, Francey LJ, Leibler JH, Asara JM, Scammell MK, Parikh SM, Brooks DR, Friedman DJ. Raines NH, et al. Among authors: leone da. Kidney Int Rep. 2024 Apr 18;9(7):2250-2259. doi: 10.1016/j.ekir.2024.04.027. eCollection 2024 Jul. Kidney Int Rep. 2024. PMID: 39081728 Free PMC article.
266 results