Title: | Where do we stand in trial readiness for autosomal recessive limb girdle muscular dystrophies? |
Journal : | Neuromuscular disorders : NMD, 26, 2 |
Authors: | Straub V, Author ; Bertoli M |
Material Type: | Article |
Publication Date: | 2016 |
Size: | p 111 |
Languages: | English |
Keywords : | animal models ; ataluren ; autosomal recessive limb girdle muscular dystrophy ; cell therapy ; chemokines ; clinical trials ; diseases classifications ; dynamometry ; exon skipping ; gene transfer ; late-onset Pompe disease ; loongitudinal study ; patient registers ; review article ; therapeutic prospects |
Keywords: | Clinical trials;LGMD2;Outcome measures;Patient registries;Translational research; |
Pubmed / DOI : | DOI : 10.1016/j.nmd.2015.11.012 / Pubmed : 26810373 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/26810373 |