Title: | Nusinersen for older patients with spinal muscular atrophy: A real-world clinical setting experience |
Journal : | Muscle & Nerve |
Authors: | Veerapandiyan A, Author ; Eichinger K ; Guntrum D ; Kwon J ; Baker L ; Collins E ; Ciafaloni E |
Material Type: | Article |
Publisher: | United States, 11/2019 |
Languages: | English |
Keywords : | adulthood ; antisense oligonucleotides ; functional assessment ; gene therapy ; motor neuron disease ; neuromuscular disease ; nusinersen ; SMN1-related proximal spinal muscular atrophy ; spinal muscular atrophy ; teenagers |
Keywords: | SMA;adolescents;adults;intrathecal;nusinersen;spinraza; |
Pubmed / DOI : | Pubmed : 31773738 / DOI : 10.1002/mus.26769 |
N° Profil MNM : | 2019121 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/31773738 |