2025
article
OpenAlex
Stephen Baine, Young-Eun Seo, A. Kempton, Oliver C. Rogers et autres
Limb-girdle muscular dystrophy type 2E/R4 (LGMD2E/R4) is an ultra-rare autosomal recessive disorder caused by mutations in SGCB , the gene that encodes for β-sarcoglycan (SGCB), a component of the dystrophin-associated protein complex that stabilizes muscle fibers during contractions. Bidridistrogene xeboparvovec is an …
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2023
article
OpenAlex
Rachael A. Potter, Chris Wier, Grace Cooper-Olson, Esther F. Wheeler et autres
Objective: The purpose of this preclinical study is to evaluate the myocardial efficacy and safety of intended commercial process delandistrogene moxeparvovec (SRP-9001) material in Duchenne muscular dystrophy (DMD)mdx rats. DMDmdx rats are a valuable alternative animal model of DMD, as they demonstrate …
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2020
article
OpenAlex
Eric R. Pozsgai, D. Griffin, Ellyn L. Peterson, A. Kempton et autres
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2020
article
OpenAlex
Eric R. Pozsgai, Ellyn L. Peterson, A. Kempton, O. Rogers et autres
us
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2020
article
OpenAlex
Young‐Joon Seo, A. Kempton, O. Rogers, S. Baine et autres
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