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A genome-wide association analysis of loss of ambulation in dystrophinopathy patients suggests multiple candidate modifiers of disease severity.
Flanigan KM, Waldrop MA, Martin PT, Alles R, Dunn DM, Alfano LN, Simmons TR, Moore-Clingenpeel M, Burian J, Seok SC, Weiss RB, Vieland VJ. Flanigan KM, et al. Among authors: martin pt. Eur J Hum Genet. 2023 Jun;31(6):663-673. doi: 10.1038/s41431-023-01329-5. Epub 2023 Mar 20. Eur J Hum Genet. 2023. PMID: 36935420
A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy using rAAVrh74.MCK.GALGT2.
Flanigan KM, Vetter TA, Simmons TR, Iammarino M, Frair EC, Rinaldi F, Chicoine LG, Harris J, Cheatham JP, Cheatham SL, Boe B, Waldrop MA, Zygmunt DA, Packer D, Martin PT. Flanigan KM, et al. Among authors: martin pt. Mol Ther Methods Clin Dev. 2022 Sep 2;27:47-60. doi: 10.1016/j.omtm.2022.08.009. eCollection 2022 Dec 8. Mol Ther Methods Clin Dev. 2022. PMID: 36186954 Free PMC article.
101 results