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A prospective observational study of associated anomalies in Hirschsprung's disease.
Orphanet J Rare Dis. 2013 Nov 23;8:184. doi: 10.1186/1750-1172-8-184.
Orphanet J Rare Dis. 2013.
PMID: 24267509
Free PMC article.
Validation of the postnatal growth and retinopathy of prematurity screening criteria: A retrospective Italian analysis.
Caruggi S, Scaramuzzi M, Calevo MG, Priolo E, Sposetti L, Camicione P, Ramenghi LA, Serafino M.
Caruggi S, et al. Among authors: sposetti l.
Eur J Ophthalmol. 2021 Apr 22:11206721211011362. doi: 10.1177/11206721211011362. Online ahead of print.
Eur J Ophthalmol. 2021.
PMID: 33887976
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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
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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.
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Correction to: Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study.
Chiriboga CA, Bruno C, Duong T, Fischer D, Mercuri E, Kirschner J, Kostera-Pruszczyk A, Jaber B, Gorni K, Kletzl H, Carruthers I, Martin C, Warren F, Scalco RS, Wagner KR, Muntoni F; JEWELFISH Study Group.
Chiriboga CA, et al.
Neurol Ther. 2023 Oct;12(5):1799-1801. doi: 10.1007/s40120-023-00503-7.
Neurol Ther. 2023.
PMID: 37395990
Free PMC article.
No abstract available.
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Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study.
Chiriboga CA, Bruno C, Duong T, Fischer D, Mercuri E, Kirschner J, Kostera-Pruszczyk A, Jaber B, Gorni K, Kletzl H, Carruthers I, Martin C, Warren F, Scalco RS, Wagner KR, Muntoni F; JEWELFISH Study Group.
Chiriboga CA, et al.
Neurol Ther. 2023 Apr;12(2):543-557. doi: 10.1007/s40120-023-00444-1. Epub 2023 Feb 13.
Neurol Ther. 2023.
PMID: 36780114
Free PMC article.
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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.
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