Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy

por Mendell, Jerry R., Al-Zaidy, Samiah A., Lehman, Kelly J., McColly, Markus, Lowes, Linda P., Alfano, Lindsay N., Reash, Natalie F., Iammarino, Megan A., Church, Kathleen R., Kleyn, Aaron, Meriggioli, Matthew N., Shell, Richard
Publicado 2021

Validation of the North Star Assessment for Limb-Girdle Type Muscular Dystrophies

por James, Meredith K, Alfano, Lindsay N, Muni-Lofra, Robert, Reash, Natalie F, Sodhi, Jassi, Iammarino, Megan A, Moat, Dionne, Shannon, Kianna, McCallum, Michelle, Richardson, Mark, Eagle, Michelle, Straub, Volker, Marini-Bettolo, Chiara, Lowes, Linda P, Mayhew, Anna G
Publicado 2022

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

Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscu...

por Mendell, Jerry R., Shieh, Perry B., McDonald, Craig M., Sahenk, Zarife, Lehman, Kelly J., Lowes, Linda P., Reash, Natalie F., Iammarino, Megan A., Alfano, Lindsay N., Sabo, Brenna, Woods, Jeremy D., Skura, Christy L., Mao, Howard C., Staudt, Loretta A., Griffin, Danielle A., Lewis, Sarah, Wang, Shufang, Potter, Rachael A., Singh, Teji, Rodino-Klapac, Louise R.
Publicado 2023