Title: | Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study |
Journal : | Journal of comparative effectiveness research |
Authors: | Mercuri E ; Muntoni F ; Osorio AN ; Tulinius M ; Buccella F ; Morgenroth LP ; Gordish Dressman H ; Jiang J ; Trifillis P ; Zhu J ; Kristensen A ; Santos CL ; Henricson EK ; McDonald CM ; Desguerre I |
Material Type: | Article |
Publisher: | England, 2020 |
Languages: | English |
Keywords : | clinical trials ; cohort studies ; drugs ; Duchenne muscular dystrophy ; muscular dystrophy ; neuromuscular disease ; pharmacotherapy |
Keywords: | STRIDE Registry;ataluren;dystrophin;effectiveness;nonsense mutation Duchenne muscular dystrophy;safety; |
Lien associé : | NCT02369731 |
Pubmed / DOI : | Pubmed : 31997646 / DOI : 10.2217/cer-2019-0171 |
N° Profil MNM : | 2020013 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/31997646 |
See also : |