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Table representation of search results timeline featuring number of search results per year.

Year Number of Results
1946 1
1967 1
1969 1
1971 1
1973 1
1974 3
1975 1
1977 1
1978 8
1979 2
1982 2
1983 4
1984 5
1985 5
1986 1
1987 1
1988 7
1989 7
1990 8
1991 6
1992 4
1993 8
1994 5
1995 6
1996 11
1997 6
1998 10
1999 11
2000 14
2001 10
2002 26
2003 20
2004 13
2005 16
2006 21
2007 19
2008 27
2009 36
2010 37
2011 35
2012 55
2013 54
2014 39
2015 63
2016 57
2017 49
2018 52
2019 54
2020 44
2021 53
2022 38
2023 7
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Search Results

859 results
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Page 1
Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Mendell JR, Al-Zaidy SA, Rodino-Klapac LR, Goodspeed K, Gray SJ, Kay CN, Boye SL, Boye SE, George LA, Salabarria S, Corti M, Byrne BJ, Tremblay JP. Mendell JR, et al. Among authors: byrne bj. Mol Ther. 2021 Feb 3;29(2):464-488. doi: 10.1016/j.ymthe.2020.12.007. Epub 2020 Dec 10. Mol Ther. 2021. PMID: 33309881 Free PMC article. Review.
Advancements in AAV-mediated Gene Therapy for Pompe Disease.
Salabarria SM, Nair J, Clement N, Smith BK, Raben N, Fuller DD, Byrne BJ, Corti M. Salabarria SM, et al. Among authors: byrne bj. J Neuromuscul Dis. 2020;7(1):15-31. doi: 10.3233/JND-190426. J Neuromuscul Dis. 2020. PMID: 31796685 Free PMC article. Review.
Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease.
Dimachkie MM, Barohn RJ, Byrne B, Goker-Alpan O, Kishnani PS, Ladha S, Laforêt P, Mengel KE, Peña LDM, Sacconi S, Straub V, Trivedi J, Van Damme P, van der Ploeg AT, Vissing J, Young P, Haack KA, Foster M, Gilbert JM, Miossec P, Vitse O, Zhou T, Schoser B; NEO-EXT investigators. Dimachkie MM, et al. Among authors: byrne b. Neurology. 2022 May 26;99(5):e536-48. doi: 10.1212/WNL.0000000000200746. Online ahead of print. Neurology. 2022. PMID: 35618441 Free PMC article.
Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study.
Pena LDM, Barohn RJ, Byrne BJ, Desnuelle C, Goker-Alpan O, Ladha S, Laforêt P, Mengel KE, Pestronk A, Pouget J, Schoser B, Straub V, Trivedi J, Van Damme P, Vissing J, Young P, Kacena K, Shafi R, Thurberg BL, Culm-Merdek K, van der Ploeg AT; NEO1 Investigator Group. Pena LDM, et al. Among authors: byrne bj. Neuromuscul Disord. 2019 Mar;29(3):167-186. doi: 10.1016/j.nmd.2018.12.004. Epub 2018 Dec 17. Neuromuscul Disord. 2019. PMID: 30770310 Free article. Clinical Trial.
Neonatal outreach simulation.
Byrne BJ, Manhas D. Byrne BJ, et al. Semin Perinatol. 2016 Nov;40(7):480-488. doi: 10.1053/j.semperi.2016.08.009. Epub 2016 Sep 28. Semin Perinatol. 2016. PMID: 27692476 Review.
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. Among authors: byrne bj. Lancet Neurol. 2021 Dec;20(12):1027-1037. doi: 10.1016/S1474-4422(21)00331-8. Lancet Neurol. 2021. PMID: 34800400 Clinical Trial.
859 results