Drug Regulation in the Era of Individualized Therapies

Affiliations

Editorial

Janet Woodcock et al. N Engl J Med. 2019.

. 2019 Oct 24;381(17):1678-1680.

doi: 10.1056/NEJMe1911295. Epub 2019 Oct 9.

¹ From the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research, Food and Drug Administration, Silver Spring, MD.

No abstract available

Comment in

Framework for "N-of-1" Experimental Therapies.
Brierley J, Aylett S, Archard D. Brierley J, et al. N Engl J Med. 2020 Jan 23;382(4):e7. doi: 10.1056/NEJMc1915778. N Engl J Med. 2020. PMID: 31971694 No abstract available.

Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease.
Kim J, Hu C, Moufawad El Achkar C, Black LE, Douville J, Larson A, Pendergast MK, Goldkind SF, Lee EA, Kuniholm A, Soucy A, Vaze J, Belur NR, Fredriksen K, Stojkovska I, Tsytsykova A, Armant M, DiDonato RL, Choi J, Cornelissen L, Pereira LM, Augustine EF, Genetti CA, Dies K, Barton B, Williams L, Goodlett BD, Riley BL, Pasternak A, Berry ER, Pflock KA, Chu S, Reed C, Tyndall K, Agrawal PB, Beggs AH, Grant PE, Urion DK, Snyder RO, Waisbren SE, Poduri A, Park PJ, Patterson A, Biffi A, Mazzulli JR, Bodamer O, Berde CB, Yu TW. Kim J, et al. N Engl J Med. 2019 Oct 24;381(17):1644-1652. doi: 10.1056/NEJMoa1813279. Epub 2019 Oct 9. N Engl J Med. 2019. PMID: 31597037 Free PMC article.