Title: | A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy |
Journal : | Neuromuscular disorders : NMD, 28, 1 |
Authors: | Goemans N, Author ; Mercuri E ; Belousova E ; Komaki H ; Dubrovsky A ; McDonald CM ; Kraus JE ; Lourbakos A ; Lin Z ; Campion G ; Wang SX ; Campbell C |
Material Type: | Article |
Publication Date: | 2018 |
Size: | p 4 |
Languages: | English |
Keywords : | 6 minute walk test ; adverse side effects ; antisense oligonucleotides ; children ; double-blind studies ; drisapersen ; drug tolerance ; Duchenne muscular dystrophy ; exon skipping ; motor skills ; phase 3 clinical trials ; randomized studies ; therapeutic efficiency |
Keywords: | Antisense oligonucleotide;Drisapersen;Duchenne muscular dystrophy;Dystrophin;Exon skipping;six-minute walking distance; |
Pubmed / DOI : | DOI : 10.1016/j.nmd.2017.10.004 / Pubmed : 29203355 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/29203355 |