Title: | Gene therapy for Duchenne muscular dystrophy: balancing good science, marginal efficacy, high emotions and excessive cost |
Journal : | Therapeutic advances in neurological disorders, 10, 8 |
Authors: | Dalakas MC, Author |
Material Type: | Article |
Publication Date: | 2017 |
Size: | p 293 |
Languages: | English |
Keywords : | bioethics ; controversy ; deflazacort ; drisapersen ; Duchenne muscular dystrophy ; eteplirsen ; exon skipping ; expenditure ; FDA (Food and drug administration) ; gene therapy ; minidystrophin |
Pubmed / DOI : | DOI : 10.1177/1756285617717155 / Pubmed : 28781610 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/28781610 |