Title: | Treatment of infantile-onset spinal muscular atrophy with nusinersen: final report of a phase 2, open-label, multicentre, dose-escalation study |
Journal : | The Lancet. Child & adolescent health, 5, 7 |
Authors: | Finkel RS ; Chiriboga CA ; Vajsar J ; Day JW ; Montes J ; de Vivo DC ; Bishop KM ; Foster R ; Liu Y ; Ramirez-Schrempp D ; Schneider E ; Bennett CF ; Wong J ; Farwell W |
Material Type: | Article |
Publisher: | England, 07/2021 |
Size: | p 491 |
Languages: | English |
Keywords : | antisense oligonucleotides ; clinical trials ; nusinersen ; phase 2 clinical trials ; SMN1-related proximal spinal muscular atrophy |
Lien associé : | NCT01839656 |
Pubmed / DOI : | Pubmed : 34089650 / DOI : 10.1016/S2352-4642(21)00100-0 |
N° Profil MNM : | 2021061 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/34089650 |