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Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodies.
J Clin Invest. 2024 Jan 2;134(1):e173510. doi: 10.1172/JCI173510.
J Clin Invest. 2024.
PMID: 37988172
Free PMC article.
Cardiac and clinical phenotype in Barth syndrome.
Spencer CT, Bryant RM, Day J, Gonzalez IL, Colan SD, Thompson WR, Berthy J, Redfearn SP, Byrne BJ.
Spencer CT, et al.
Pediatrics. 2006 Aug;118(2):e337-46. doi: 10.1542/peds.2005-2667. Epub 2006 Jul 17.
Pediatrics. 2006.
PMID: 16847078
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Polysomnography findings in children with spinal muscular atrophy after onasemnogene-abeparvovec.
Leon-Astudillo C, Wagner M, Salabarria SM, Lammers J, Berthy J, Zingariello CD, Byrne BJ, Smith BK.
Leon-Astudillo C, et al.
Sleep Med. 2023 Jan;101:234-237. doi: 10.1016/j.sleep.2022.11.006. Epub 2022 Nov 10.
Sleep Med. 2023.
PMID: 36442421
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Perspectives of the Friedreich ataxia community on gene therapy clinical trials.
Trantham SJ, Coker MA, Norman S, Crowley E, Berthy J, Byrne BJ, Subramony S, Lou X, Corti M.
Trantham SJ, et al.
Mol Ther Methods Clin Dev. 2023 Dec 18;32(1):101179. doi: 10.1016/j.omtm.2023.101179. eCollection 2024 Mar 14.
Mol Ther Methods Clin Dev. 2023.
PMID: 38261944
Free PMC article.
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Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial.
Schoser B, Roberts M, Byrne BJ, Sitaraman S, Jiang H, Laforêt P, Toscano A, Castelli J, Díaz-Manera J, Goldman M, van der Ploeg AT, Bratkovic D, Kuchipudi S, Mozaffar T, Kishnani PS; PROPEL Study Group.
Schoser B, et al.
Lancet Neurol. 2021 Dec;20(12):1027-1037. doi: 10.1016/S1474-4422(21)00331-8.
Lancet Neurol. 2021.
PMID: 34800400
Clinical Trial.
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104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07).
Schoser B, Kishnani PS, Bratkovic D, Byrne BJ, Claeys KG, Díaz-Manera J, Laforêt P, Roberts M, Toscano A, van der Ploeg AT, Castelli J, Goldman M, Holdbrook F, Sitaraman Das S, Wasfi Y, Mozaffar T; ATB200-07 Study Group.
Schoser B, et al.
J Neurol. 2024 May;271(5):2810-2823. doi: 10.1007/s00415-024-12236-0. Epub 2024 Feb 28.
J Neurol. 2024.
PMID: 38418563
Free PMC article.
Clinical Trial.
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