A phase 2 open-label study of the safety and efficacy of weekly dosing of ATL1102 in patients with non-ambulatory Duchenne muscular dystrophy and pharmacology in mdx mice
一项针对非行走型杜氏肌营养不良症患者每周给药的ATL1102的安全性和有效性的II期开放标签研究,以及在mdx小鼠中的药理学研究。
期刊:PLoS One
影响因子:2.6
doi:10.1371/journal.pone.0294847
Woodcock, Ian R; Tachas, George; Desem, Nuket; Houweling, Peter J; Kean, Michael; Emmanuel, Jaiman; Kennedy, Rachel; Carroll, Kate; de Valle, Katy; Adams, Justine; Lamandé, Shireen R; Coles, Chantal; Tiong, Chrystal; Burton, Matthew; Villano, Daniella; Button, Peter; Hogrel, Jean-Yves; Catling-Seyffer, Sarah; Ryan, Monique M; Delatycki, Martin B; Yiu, Eppie M