Title: | The SMA Trust: the role of a disease-focused research charity in developing treatments for SMA |
Journal : | Gene therapy, 24, 9 |
Authors: | Christie-Brown V, Author ; Mitchell J ; Talbot K |
Material Type: | Article |
Publication Date: | 2017 |
Size: | p 544 |
Languages: | English |
Keywords : | associations ; nusinersen ; SMA Trust ; SMN1-related proximal spinal muscular atrophy ; therapeutic prospects ; United Kingdom |
Pubmed / DOI : | DOI : 10.1038/gt.2017.47 / Pubmed : 28561814 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/28561814 |