A Roadmap to Newborn Screening for Duchenne Muscular Dystrophy

por Al-Zaidy, Samiah A., Lloyd-Puryear, Michele, Kennedy, Annie, Lopez, Veronica, Mendell, Jerry R.
Publicado 2017

Follistatin Gene Therapy Improves Ambulation in Becker Muscular Dystrophy

por Al-Zaidy, Samiah A., Sahenk, Zarife, Rodino-Klapac, Louise R., Kaspar, Brian, Mendell, Jerry R.
Publicado 2015

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

Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy

por Al‐Zaidy, Samiah, Pickard, A. Simon, Kotha, Kavitha, Alfano, Lindsay N., Lowes, Linda, Paul, Grace, Church, Kathleen, Lehman, Kelly, Sproule, Douglas M., Dabbous, Omar, Maru, Benit, Berry, Katherine, Arnold, W. David, Kissel, John T., Mendell, Jerry R., Shell, Richard
Publicado 2018

Current Clinical Applications of In Vivo Gene Therapy with AAVs

por Mendell, Jerry R., Al-Zaidy, Samiah A., Rodino-Klapac, Louise R., Goodspeed, Kimberly, Gray, Steven J., Kay, Christine N., Boye, Sanford L., Boye, Shannon E., George, Lindsey A., Salabarria, Stephanie, Corti, Manuela, Byrne, Barry J., Tremblay, Jacques P.
Publicado 2021

A slowly progressive form of limb-girdle muscular dystrophy type 2C associated with founder mutation in the SGCG gene in Puerto Rican Hispanics

por Al-Zaidy, Samiah A, Malik, Vinod, Kneile, Kelley, Rosales, Xiomara Q, Gomez, Ana Maria, Lewis, Sarah, Hashimoto, Sayaka, Gastier-Foster, Julie, Kang, Peter, Darras, Basil, Kunkel, Louis, Carlo, Jose, Sahenk, Zarife, Moore, Steven A, Pyatt, Robert, Mendell, Jerry R
Publicado 2015

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

Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

por Mendell, Jerry R., Goemans, Nathalie, Lowes, Linda P., Alfano, Lindsay N., Berry, Katherine, Shao, James, Kaye, Edward M., Mercuri, Eugenio, Hamid, Hoda Abdel, Byrne, Barry J., Connolly, Anne M., Dracker, Robert A., Matthew Frank, L., Heydemann, Peter T., O'Brien, Kevin C., Sparks, Susan E., Specht, Linda A., Rodino‐Klapac, Louise, Sahenk, Zarife, Al‐Zaidy, Samiah, Cripe, Linda H., Lewis, Sarah, M, Pane, E, Mazzone, S, Messina, GL, Vita, Bertini, D Amico A, Casimiro, Berardinelli A, Y, Torrente, F, Magri, GP, Comi, G, Baranello, T, Mongini, A, Pini, R, Battini, E, Pegoraro, C, Bruno, L, Politano, S, Previtali
Publicado 2016