Title: | Therapeutic approaches for spinal muscular atrophy (SMA) |
Journal : | Gene therapy, 24, 9 |
Authors: | Scoto M, Author ; Finkel RS ; Mercuri E ; Muntoni F |
Material Type: | Article |
Publication Date: | 2017 |
Size: | p 514 |
Languages: | English |
Keywords : | clinical trials ; gene therapy ; medical treatment ; nusinersen ; olesoxime ; reviews ; salbutamol ; SMN protein ; SMN1 gene ; SMN1-related proximal spinal muscular atrophy ; SMN2 gene ; splicing ; stem cells ; therapeutic prospects ; troponin |
Pubmed / DOI : | DOI : 10.1038/gt.2017.45 / Pubmed : 28561813 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/28561813 |