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

Expression and function of four AAV-based constructs for dystrophin restoration in the mdx mouse model of Duchenne muscular dystrophy

por Potter, Rachael A., Griffin, Danielle A., Heller, Kristin N., Mendell, Jerry R., Rodino-Klapac, Louise R.
Publicado 2023

Long-Term Pulmonary Function in Duchenne Muscular Dystrophy: Comparison of Eteplirsen-Treated Patients to Natural History

por Kinane, T. Bernard, Mayer, Oscar H., Duda, Petra W., Lowes, Linda P., Moody, Stephanie L., Mendell, Jerry R.
Publicado 2018

Adeno-associated virus serotype 9 antibody seroprevalence for patients in the United States with spinal muscular atrophy

por Day, John W., Mendell, Jerry R., Burghes, Arthur H.M., van Olden, Rudolf W., Adhikary, Rishi R., Dilly, Keith W.
Publicado 2023

Assessment of rAAVrh.74.MHCK7.micro-dystrophin Gene Therapy Using Magnetic Resonance Imaging in Children With Duchenne Muscular Dystrophy

por Willcocks, Rebecca J., Forbes, Sean C., Walter, Glenn A., Sweeney, Lee, Rodino-Klapac, Louise R., Mendell, Jerry R., Vandenborne, Krista
Publicado 2021

Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the phenotype in a mouse model for LGMD2A/R1

por Sahenk, Zarife, Ozes, Burcak, Murrey, Darren, Myers, Morgan, Moss, Kyle, Yalvac, Mehmet E., Ridgley, Alicia, Chen, Lei, Mendell, Jerry R.
Publicado 2021

Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec

por Day, John W., Finkel, Richard S., Mercuri, Eugenio, Swoboda, Kathryn J., Menier, Melissa, van Olden, Rudolf, Tauscher-Wisniewski, Sitra, Mendell, Jerry R.
Publicado 2021

A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy

por Rodino-Klapac, Louise R, Janssen, Paul ML, Montgomery, Chrystal L, Coley, Brian D, Chicoine, Louis G, Clark, K Reed, Mendell, Jerry R
Publicado 2007

Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the mdx Mouse Model of Duchenne Muscular Dystrophy

por Potter, Rachael A., Griffin, Danielle A., Heller, Kristin N., Peterson, Ellyn L., Clark, Emma K., Mendell, Jerry R., Rodino-Klapac, Louise R.
Publicado 2021

Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United States

por Klimchak, Alexa C., Sedita, Lauren E., Rodino-Klapac, Louise R., Mendell, Jerry R., McDonald, Craig M., Gooch, Katherine L., Malone, Daniel C.
Publicado 2023

Comparison of Long-term Ambulatory Function in Patients with Duchenne Muscular Dystrophy Treated with Eteplirsen and Matched Natural History Controls

por Mendell, Jerry R., Khan, Navid, Sha, Nanshi, Eliopoulos, Helen, McDonald, Craig M., Goemans, Nathalie, Mercuri, Eugenio, Lowes, Linda P., Alfano, Lindsay N.
Publicado 2021

Cardiovascular magnetic resonance of cardiomyopathy in limb girdle muscular dystrophy 2B and 2I

por Rosales, Xiomara Q, Moser, Sean J, Tran, Tam, McCarthy, Beth, Dunn, Nicholas, Habib, Philip, Simonetti, Orlando P, Mendell, Jerry R, Raman, Subha V
Publicado 2011

Proof of Concept of the Ability of the Kinect to Quantify Upper Extremity Function in Dystrophinopathy

por Lowes, Linda P, Alfano, Lindsay N, Yetter, Brent A, Worthen-Chaudhari, Lise, Hinchman, William, Savage, Jordan, Samona, Patrick, Flanigan, Kevin M, Mendell, Jerry R
Publicado 2013

Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy

por Khan, Navid, Eliopoulos, Helen, Han, Lixin, Kinane, T. Bernard, Lowes, Linda P., Mendell, Jerry R., Gordish-Dressman, Heather, Henricson, Erik K., McDonald, Craig M.
Publicado 2019

AAV1.NT-3 Gene Therapy for Charcot–Marie–Tooth Neuropathy

por Sahenk, Zarife, Galloway, Gloria, Clark, Kelly Reed, Malik, Vinod, Rodino-Klapac, Louise R, Kaspar, Brian K., Chen, Lei, Braganza, Cilwyn, Montgomery, Chrystal, Mendell, Jerry R
Publicado 2014

Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations

por Mendell, Jerry R., Connolly, Anne M., Lehman, Kelly J., Griffin, Danielle A., Khan, Sohrab Z., Dharia, Sachi D., Quintana-Gallardo, Lucía, Rodino-Klapac, Louise R.
Publicado 2022

AAV1.NT-3 gene therapy for X-linked Charcot–Marie–Tooth neuropathy type 1

por Ozes, Burcak, Myers, Morgan, Moss, Kyle, Mckinney, Jennifer, Ridgley, Alicia, Chen, Lei, Bai, Shasha, Abrams, Charles K., Freidin, Mona M., Mendell, Jerry R., Sahenk, Zarife
Publicado 2021

Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy

por Day, John W., Mendell, Jerry R., Mercuri, Eugenio, Finkel, Richard S., Strauss, Kevin A., Kleyn, Aaron, Tauscher-Wisniewski, Sitra, Tukov, Francis Fonyuy, Reyna, Sandra P., Chand, Deepa H.
Publicado 2021

Correction to: Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy

por Day, John W., Mendell, Jerry R., Mercuri, Eugenio, Finkel, Richard S., Strauss, Kevin A., Kleyn, Aaron, Tauscher-Wisniewski, Sitra, Tukov, Francis Fonyuy, Reyna, Sandra P., Chand, Deepa H.
Publicado 2021