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2014 3
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2017 2
2018 1
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2022 4
2024 0

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Page 1
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.
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-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls.
Darras BT, Masson R, Mazurkiewicz-Bełdzińska M, Rose K, Xiong H, Zanoteli E, Baranello G, Bruno C, Vlodavets D, Wang Y, El-Khairi M, Gerber M, Gorni K, Khwaja O, Kletzl H, Scalco RS, Fontoura P, Servais L; FIREFISH Working Group. Darras BT, et al. N Engl J Med. 2021 Jul 29;385(5):427-435. doi: 10.1056/NEJMoa2102047. N Engl J Med. 2021. PMID: 34320287
Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial.
Mercuri E, Deconinck N, Mazzone ES, Nascimento A, Oskoui M, Saito K, Vuillerot C, Baranello G, Boespflug-Tanguy O, Goemans N, Kirschner J, Kostera-Pruszczyk A, Servais L, Gerber M, Gorni K, Khwaja O, Kletzl H, Scalco RS, Staunton H, Yeung WY, Martin C, Fontoura P, Day JW; SUNFISH Study Group. Mercuri E, et al. Lancet Neurol. 2022 Jan;21(1):42-52. doi: 10.1016/S1474-4422(21)00367-7. Lancet Neurol. 2022. PMID: 34942136
Outcome measures for children with movement disorders.
Pagliano E, Baranello G, Masson R, Foscan M, Arnoldi MT, Marchi A, Aprile G, Pantaleoni C. Pagliano E, et al. Among authors: arnoldi mt. Eur J Paediatr Neurol. 2018 May;22(3):346-353. doi: 10.1016/j.ejpn.2018.01.014. Epub 2018 Feb 8. Eur J Paediatr Neurol. 2018. PMID: 29475818 Review.
Functional outcome measures in young, steroid-naïve boys with Duchenne muscular dystrophy.
Mayhew AG, Moat D, McDermott MP, Eagle M, Griggs RC, King WM, James MK, Muni-Lofra R, Shillington A, Gregson S, Pallant L, Skura C, Staudt LA, Eichinger K, McMurchie H, Rabb R, Di Marco M, Brown S, Zanin R, Arnoldi MT, McIntyre M, Wilson A, Alfano LN, Lowes LP, Blomgren C, Milev E, Iodice M, Pasternak A, Chiu A, Lehnert I, Claus N, Dieruf KA, Rolle E, Nicorici A, Andres B, Hobbiebrunken E, Roetmann G, Kern V, Civitello M, Vogt S, Hayes MJ, Scholtes C, Lacroix C, Gunn T, Warner S, Newman J, Barp A, Kundrat K, Kovelman S, Powers PJ, Guglieri M; Muscle Study Group and TREAT-NMD. Mayhew AG, et al. Among authors: arnoldi mt. Neuromuscul Disord. 2022 Jun;32(6):460-467. doi: 10.1016/j.nmd.2022.02.012. Epub 2022 Feb 26. Neuromuscul Disord. 2022. PMID: 35618576 Free article.
Sleep disorders in spinal muscular atrophy.
Pera MC, Romeo DM, Graziano A, Palermo C, Messina S, Baranello G, Coratti G, Massaro M, Sivo S, Arnoldi MT, Mazzone ES, Antonaci L, Lapenta L, Albamonte E, Fanelli L, de Sanctis R, Vita GL, Sframeli M, Pane M, Mercuri E. Pera MC, et al. Among authors: arnoldi mt. Sleep Med. 2017 Feb;30:160-163. doi: 10.1016/j.sleep.2016.11.012. Epub 2016 Nov 30. Sleep Med. 2017. PMID: 28215241
Bone and Spinal Muscular Atrophy.
Vai S, Bianchi ML, Moroni I, Mastella C, Broggi F, Morandi L, Arnoldi MT, Bussolino C, Baranello G. Vai S, et al. Among authors: arnoldi mt. Bone. 2015 Oct;79:116-20. doi: 10.1016/j.bone.2015.05.039. Epub 2015 Jun 5. Bone. 2015. PMID: 26055105
Evaluation of body composition as a potential biomarker in spinal muscular atrophy.
Baranello G, De Amicis R, Arnoldi MT, Zanin R, Mastella C, Masson R, Leone A, Alberti K, Foppiani A, Battezzati A, Bertoli S. Baranello G, et al. Among authors: arnoldi mt. Muscle Nerve. 2020 Apr;61(4):530-534. doi: 10.1002/mus.26823. Epub 2020 Feb 7. Muscle Nerve. 2020. PMID: 32012296 Free article.
Longitudinal natural history in young boys with Duchenne muscular dystrophy.
Coratti G, Brogna C, Norcia G, Ricotti V, Abbott L, D'Amico A, Berardinelli A, Vita GL, Lucibello S, Messina S, Sansone V, Albamonte E, Colia G, Salmin F, Gardani A, Manzur A, Main M, Baranello G, Arnoldi MT, Parsons J, Carry T, Connolly AM, Bertini E, Muntoni F, Pane M, Mercuri E. Coratti G, et al. Among authors: arnoldi mt. Neuromuscul Disord. 2019 Nov;29(11):857-862. doi: 10.1016/j.nmd.2019.09.010. Epub 2019 Sep 26. Neuromuscul Disord. 2019. PMID: 31629611
22 results