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Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy.
Conrado DJ, Larkindale J, Berg A, Hill M, Burton J, Abrams KR, Abresch RT, Bronson A, Chapman D, Crowther M, Duong T, Gordish-Dressman H, Harnisch L, Henricson E, Kim S, McDonald CM, Schmidt S, Vong C, Wang X, Wong BL, Yong F, Romero K; Duchenne Muscular Dystrophy Regulatory Science Consortium (D-RSC). Conrado DJ, et al. Among authors: yong f. J Pharmacokinet Pharmacodyn. 2019 Oct;46(5):441-455. doi: 10.1007/s10928-019-09642-7. Epub 2019 May 24. J Pharmacokinet Pharmacodyn. 2019. PMID: 31127458
Dystrophin and mini-dystrophin quantification by mass spectrometry in skeletal muscle for gene therapy development in Duchenne muscular dystrophy.
Farrokhi V, Walsh J, Palandra J, Brodfuehrer J, Caiazzo T, Owens J, Binks M, Neelakantan S, Yong F, Dua P, Le Guiner C, Neubert H. Farrokhi V, et al. Among authors: yong f. Gene Ther. 2022 Nov;29(10-11):608-615. doi: 10.1038/s41434-021-00300-7. Epub 2021 Nov 5. Gene Ther. 2022. PMID: 34737451 Free PMC article.
172 results