Title: | New treatments in spinal muscular atrophy: an overview of currently available data |
Journal : | Expert opinion on pharmacotherapy, 21, 3 |
Authors: | Ramdas S ; Servais L |
Material Type: | Article |
Publisher: | England, 02/2020 |
Size: | p 307 |
Languages: | English |
Keywords : | gene therapy ; motor neuron disease ; neuromuscular disease ; pharmacotherapy ; review article ; SMN1-related proximal spinal muscular atrophy ; spinal muscular atrophy ; therapeutic strategy |
Keywords: | spinal muscular atrophy;antisense oligonucleotide;branaplam;gene therapy;nusinersen;risdiplam;splicing modifiers; |
Pubmed / DOI : | Pubmed : 31973611 / DOI : 10.1080/14656566.2019.1704732 |
N° Profil MNM : | 2020013 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/31973611 |