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

Year Number of Results
1984 1
1987 1
1992 1
1993 2
1994 2
1995 3
1996 3
1997 8
1998 7
1999 9
2000 2
2001 5
2002 7
2003 3
2004 3
2005 1
2006 2
2007 2
2008 2
2010 3
2011 5
2012 6
2013 9
2014 13
2015 8
2016 8
2017 14
2018 6
2019 18
2020 11
2021 12
2022 11
2023 9
2024 4

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187 results

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Page 1
A framework for individualized splice-switching oligonucleotide therapy.
Kim J, Woo S, de Gusmao CM, Zhao B, Chin DH, DiDonato RL, Nguyen MA, Nakayama T, Hu CA, Soucy A, Kuniholm A, Thornton JK, Riccardi O, Friedman DA, El Achkar CM, Dash Z, Cornelissen L, Donado C, Faour KNW, Bush LW, Suslovitch V, Lentucci C, Park PJ, Lee EA, Patterson A, Philippakis AA, Margus B, Berde CB, Yu TW. Kim J, et al. Among authors: yu tw. Nature. 2023 Jul;619(7971):828-836. doi: 10.1038/s41586-023-06277-0. Epub 2023 Jul 12. Nature. 2023. PMID: 37438524 Free PMC article.
Moving away from one disease at a time: Screening, trial design, and regulatory implications of novel platform technologies.
Lekstrom-Himes J, Brooks PJ, Koeberl DD, Brower A, Goldenberg A, Green RC, Morris JA, Orsini JJ, Yu TW, Augustine EF. Lekstrom-Himes J, et al. Among authors: yu tw. Am J Med Genet C Semin Med Genet. 2023 Mar;193(1):30-43. doi: 10.1002/ajmg.c.32031. Epub 2023 Feb 4. Am J Med Genet C Semin Med Genet. 2023. PMID: 36738469 Free PMC article.
Are we prepared to deliver gene-targeted therapies for rare diseases?
Yu TW, Kingsmore SF, Green RC, MacKenzie T, Wasserstein M, Caggana M, Gold NB, Kennedy A, Kishnani PS, Might M, Brooks PJ, Morris JA, Parisi MA, Urv TK. Yu TW, et al. Am J Med Genet C Semin Med Genet. 2023 Mar;193(1):7-12. doi: 10.1002/ajmg.c.32029. Epub 2023 Jan 24. Am J Med Genet C Semin Med Genet. 2023. PMID: 36691939
Data sharing to advance gene-targeted therapies in rare diseases.
Lekstrom-Himes J, Augustine EF, Brower A, Defay T, Finkel RS, McGuire AL, Skinner MW, Yu TW. Lekstrom-Himes J, et al. Among authors: yu tw. Am J Med Genet C Semin Med Genet. 2023 Mar;193(1):87-98. doi: 10.1002/ajmg.c.32028. Epub 2023 Jan 3. Am J Med Genet C Semin Med Genet. 2023. PMID: 36594517
187 results