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

Year Number of Results
2009 1
2014 3
2015 5
2016 4
2017 3
2018 7
2019 5
2020 5
2021 7
2022 7
2023 5
2024 2

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44 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.
First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B.
Harmatz P, Prada CE, Burton BK, Lau H, Kessler CM, Cao L, Falaleeva M, Villegas AG, Zeitler J, Meyer K, Miller W, Wong Po Foo C, Vaidya S, Swenson W, Shiue LH, Rouy D, Muenzer J. Harmatz P, et al. Among authors: lau h. Mol Ther. 2022 Dec 7;30(12):3587-3600. doi: 10.1016/j.ymthe.2022.10.010. Epub 2022 Oct 25. Mol Ther. 2022. PMID: 36299240 Free PMC article.
Application of CRISPR genetic screens to investigate neurological diseases.
So RWL, Chung SW, Lau HHC, Watts JJ, Gaudette E, Al-Azzawi ZAM, Bishay J, Lin LT, Joung J, Wang X, Schmitt-Ulms G. So RWL, et al. Among authors: lau hhc. Mol Neurodegener. 2019 Nov 14;14(1):41. doi: 10.1186/s13024-019-0343-3. Mol Neurodegener. 2019. PMID: 31727120 Free PMC article. Review.
Efficacy and Safety of N-Acetyl-l-Leucine in Children and Adults With GM2 Gangliosidoses.
Martakis K, Claassen J, Gascon-Bayari J, Goldschagg N, Hahn A, Hassan A, Hennig A, Jones S, Kay R, Lau H, Perlman S, Sharma R, Schneider S, Bremova-Ertl T. Martakis K, et al. Among authors: lau h. Neurology. 2023 Mar 7;100(10):e1072-e1083. doi: 10.1212/WNL.0000000000201660. Epub 2022 Dec 1. Neurology. 2023. PMID: 36456200 Free PMC article. Clinical Trial.
Aceruloplasminemia and putaminal cavitation.
Riboldi GM, Anstett K, Jain R, Lau H, Swope D. Riboldi GM, et al. Among authors: lau h. Parkinsonism Relat Disord. 2018 Jun;51:121-123. doi: 10.1016/j.parkreldis.2018.03.003. Epub 2018 Mar 7. Parkinsonism Relat Disord. 2018. PMID: 29534945 No abstract available.
Alpha-synuclein seeding shows a wide heterogeneity in multiple system atrophy.
Martinez-Valbuena I, Visanji NP, Kim A, Lau HHC, So RWL, Alshimemeri S, Gao A, Seidman MA, Luquin MR, Watts JC, Lang AE, Kovacs GG. Martinez-Valbuena I, et al. Among authors: lau hhc. Transl Neurodegener. 2022 Feb 7;11(1):7. doi: 10.1186/s40035-022-00283-4. Transl Neurodegener. 2022. PMID: 35125105 Free PMC article.
Delivery of epilepsy care to adults with intellectual and developmental disabilities.
Devinsky O, Asato M, Camfield P, Geller E, Kanner AM, Keller S, Kerr M, Kossoff EH, Lau H, Kothare S, Singh BK, Wirrell E. Devinsky O, et al. Among authors: lau h. Neurology. 2015 Oct 27;85(17):1512-21. doi: 10.1212/WNL.0000000000002060. Epub 2015 Sep 30. Neurology. 2015. PMID: 26423430 Free PMC article. Review.
44 results