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

Year Number of Results
1989 2
1990 1
1991 4
1992 2
1993 2
1994 4
1995 5
1996 4
1997 6
1998 1
1999 5
2000 15
2001 5
2002 7
2003 10
2004 4
2005 8
2006 20
2007 28
2008 20
2009 24
2010 20
2011 18
2012 18
2013 22
2014 22
2015 19
2016 16
2017 17
2018 10
2019 9
2020 15
2021 7
2022 8
2023 17
2024 5

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356 results

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Page 1
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.
Niemann-Pick disease type C.
Vanier MT. Vanier MT. Orphanet J Rare Dis. 2010 Jun 3;5:16. doi: 10.1186/1750-1172-5-16. Orphanet J Rare Dis. 2010. PMID: 20525256 Free PMC article. Review.
Metabolic Control of Astrocyte Pathogenic Activity via cPLA2-MAVS.
Chao CC, Gutiérrez-Vázquez C, Rothhammer V, Mayo L, Wheeler MA, Tjon EC, Zandee SEJ, Blain M, de Lima KA, Takenaka MC, Avila-Pacheco J, Hewson P, Liu L, Sanmarco LM, Borucki DM, Lipof GZ, Trauger SA, Clish CB, Antel JP, Prat A, Quintana FJ. Chao CC, et al. Cell. 2019 Dec 12;179(7):1483-1498.e22. doi: 10.1016/j.cell.2019.11.016. Epub 2019 Dec 5. Cell. 2019. PMID: 31813625 Free PMC article.
Cipaglucosidase Alfa: First Approval.
Blair HA. Blair HA. Drugs. 2023 Jun;83(8):739-745. doi: 10.1007/s40265-023-01886-5. Drugs. 2023. PMID: 37184753 Free PMC article. Review.
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.
356 results