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

Year Number of Results
2002 2
2006 2
2007 1
2009 2
2010 1
2011 1
2012 3
2013 5
2014 1
2015 2
2016 5
2017 5
2018 2
2019 1
2020 3
2021 1
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32 results
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Page 1
[SMA: from gene discovery to gene therapy].
Barkats M. Barkats M. Med Sci (Paris). 2020 Feb;36(2):137-140. doi: 10.1051/medsci/2020010. Epub 2020 Mar 4. Med Sci (Paris). 2020. PMID: 32129749 Free article. Review. French.
Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.
Puzzo F, Colella P, Biferi MG, Bali D, Paulk NK, Vidal P, Collaud F, Simon-Sola M, Charles S, Hardet R, Leborgne C, Meliani A, Cohen-Tannoudji M, Astord S, Gjata B, Sellier P, van Wittenberghe L, Vignaud A, Boisgerault F, Barkats M, Laforet P, Kay MA, Koeberl DD, Ronzitti G, Mingozzi F. Puzzo F, et al. Among authors: barkats m. Sci Transl Med. 2017 Nov 29;9(418):eaam6375. doi: 10.1126/scitranslmed.aam6375. Sci Transl Med. 2017. PMID: 29187643 Free PMC article.
Moving towards treatments for spinal muscular atrophy: hopes and limits.
Wirth B, Barkats M, Martinat C, Sendtner M, Gillingwater TH. Wirth B, et al. Among authors: barkats m. Expert Opin Emerg Drugs. 2015 Sep;20(3):353-6. doi: 10.1517/14728214.2015.1041375. Epub 2015 Jul 3. Expert Opin Emerg Drugs. 2015. PMID: 25920617 Review.
Systemic Treatment of Fabry Disease Using a Novel AAV9 Vector Expressing α-Galactosidase A.
Biferi MG, Cohen-Tannoudji M, García-Silva A, Souto-Rodríguez O, Viéitez-González I, San-Millán-Tejado B, Fernández-Carrera A, Pérez-Márquez T, Teijeira-Bautista S, Barrera S, Domínguez V, Marais T, González-Fernández Á, Barkats M, Ortolano S. Biferi MG, et al. Among authors: barkats m. Mol Ther Methods Clin Dev. 2020 Oct 22;20:1-17. doi: 10.1016/j.omtm.2020.10.016. eCollection 2021 Mar 12. Mol Ther Methods Clin Dev. 2020. PMID: 33335943 Free PMC article.
32 results