Title: | Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study |
Journal : | Lancet (London, England), 388, 10063 |
Authors: | Finkel RS ; Chiriboga CA ; Vajsar J ; Day JW ; Montes J ; de Vivo DC ; Yamashita M ; Rigo F ; Hung G ; Schneider E ; Norris DA ; Xia S ; Bennett CF ; Bishop KM |
Material Type: | Article |
Publication Date: | 2016 |
Size: | p 3017 |
Languages: | English |
Keywords : | compassionate use of drugs in France ; drug tolerance ; infants ; motor skills ; nusinersen ; phase 2 clinical trials ; phase 3 clinical trials ; proximal spinal muscular atrophy type 1 ; SMN1-related proximal spinal muscular atrophy |
Abstract: |
Comment in:
Dawn of a new therapeutic era for spinal muscular atrophy. [Lancet. 2016] Motor neuron disease: Nusinersen potentially effective in SMA. [Nat Rev Neurol. 2017] Why antisense could make sense for neurodegeneration. [J Neurol. 2017] |
Pubmed / DOI : | DOI : 10.1016/S0140-6736(16)31408-8 / Pubmed : 27939059 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/27939059 |
See also : |