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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: morris ja. 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. Among authors: morris ja. 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
727 results