Duchenne muscular dystrophy

Flanigan, K., Vetter, T., Simmons, T., Iammarino, M., Frair, E., Rinaldi, F., … Martin, P. (2022). A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy using rAAVrh74.MCK.GALGT2. Molecular Therapy. Methods & Clinical Development, 27, 47-60. https://doi.org/10.1016/j.omtm.2022.08.009 (Original work published 2022)

Anthony, K., Ala, P., Catapano, F., Meng, J., Domingos, J., Perry, M., … Muntoni, F. (2023). T Cell Responses to Dystrophin in a Natural History Study of Duchenne Muscular Dystrophy. Human Gene Therapy, 34(9-10), 439-448. https://doi.org/10.1089/hum.2022.166 (Original work published 2023)