Title: | Combination therapy with nusinersen and AVXS-101 in SMA type 1 |
Journal : | Neurology |
Authors: | Lee BH, Author ; Collins E ; Lewis L ; Guntrum D ; Eichinger K ; Voter K ; Abdel Hamid HZ ; Ciafaloni E |
Material Type: | Article |
Publisher: | United States, 09/2019 |
Languages: | English |
Keywords : | combined therapy ; gene therapy ; motor neuron disease ; neuromuscular disease ; nusinersen ; observational research ; onasemnogene abeparvovec-xioi ; SMN1-related proximal spinal muscular atrophy |
Pubmed / DOI : | Pubmed : 31488615 / DOI : 10.1212/WNL.0000000000008207 |
N° Profil MNM : | 2019092 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/31488615 |
See also : |