Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial

por Mendell, Jerry R., Sahenk, Zarife, Lehman, Kelly, Nease, Carrie, Lowes, Linda P., Miller, Natalie F., Iammarino, Megan A., Alfano, Lindsay N., Nicholl, Amanda, Al-Zaidy, Samiah, Lewis, Sarah, Church, Kathleen, Shell, Richard, Cripe, Linda H., Potter, Rachael A., Griffin, Danielle A., Pozsgai, Eric, Dugar, Ashish, Hogan, Mark, Rodino-Klapac, Louise R.
Publicado 2020