Title: | Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with external controls |
Journal : | Neuromuscular disorders : NMD, 32, 4 |
Authors: | Muntoni F ; Signorovitch J ; Sajeev G ; Goemans N ; Wong B ; Tian C ; Mercuri E ; Done N ; Wong H ; Moss J ; Yao Z ; Ward SJ ; Manzur A ; Servais L ; Niks EH ; Straub V ; de Groot IJ ; McDonald C |
Material Type: | Article |
Publisher: | England, 04/2022 |
Size: | p 271 |
Languages: | English |
Keywords : | clinical trials ; Duchenne muscular dystrophy ; information ; pharmacology |
Keywords: | Clinical trials;Drug evaluation;Duchenne muscular dystrophy;External controls;Natural history data;Real-world data; |
Pubmed / DOI : | Pubmed : 35396092 / DOI : 10.1016/j.nmd.2022.02.009 |
N° Profil MNM : | 2022042 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/35396092 |
See also : |