Title: | Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy |
Journal : | Pediatric pulmonology, 54, 2 |
Authors: | Al Zaidy S, Author ; Pickard AS ; Kotha K ; Alfano LN ; Lowes L ; Paul G ; Church K ; Lehman K ; Sproule DM ; Dabbous O ; Maru B ; Berry K ; Arnold WD ; Kissel JT ; Mendell JR ; Shell R |
Material Type: | Article |
Publication Date: | 02/2019 |
Size: | p 179 |
Languages: | English |
Keywords: | AVXS-101; SMA1; gene replacement; gene therapy; health outcomes; quality of life; spinal muscular atrophy |
Pubmed / DOI : | DOI : 10.1002/ppul.24203 / Pubmed : 30548438 |
N° Profil MNM : | 2018122 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/30548438 |
See also : |