Title: | Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial |
Journal : | JAMA Neurology |
Authors: | Mendell JR ; Sahenk Z ; Lehman K ; Nease C ; Lowes LP ; Miller NF ; Iammarino MA ; Alfano LN ; Nicholl A ; Al Zaidy S ; Lewis S ; Church K ; Shell R ; Cripe LH ; Potter RA ; Griffin DA ; Pozsgai E ; Dugar A ; Hogan M ; Rodino-Klapac LR |
Material Type: | Article |
Publication Date: | 06/2020 |
Languages: | English |
Keywords : | clinical trials ; delandistrogene moxeparvovec-rokl ; Duchenne muscular dystrophy ; gene therapy ; microdystrophin ; phase 1/2 clinical trials |
Lien associé : | NCT03375164 |
Pubmed / DOI : | Pubmed : 32539076 / DOI : 10.1001/jamaneurol.2020.1484 |
N° Profil MNM : | 2020061 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/32539076 |
See also : |