Title: | Nusinersen in later-onset spinal muscular atrophy : Long-term results from the phase 1/2 studies |
Journal : | Neurology, 92 |
Authors: | Darras BT, Author ; Chiriboga CA, Author ; Iannaccone ST, Author ; Swoboda KJ, Author ; Montes J, Author ; Xia S, Author ; Bennett FC, Author ; Bishop KM, Author ; Shefner JM, Author ; Gheuens S, Author ; Schneider E, Author ; Farwell W, Author ; de Vivo DC, Author |
Material Type: | Article |
Publication Date: | 2019 |
Size: | e2492 |
Languages: | English |
Keywords : | antisense oligonucleotides ; children ; clinical trials ; nusinersen ; proximal spinal muscular atrophy type 2 ; proximal spinal muscular atrophy type 3 ; SMN1-related proximal spinal muscular atrophy ; spinal muscular atrophy |
Lien associé : |
ClinicalTrials.gov NCT01703988 ClinicalTrials.gov NCT02052791 Texte complet disponible sur PubMed Central (PMC) |
Pubmed / DOI : | Pubmed : 31019106 / DOI : 10.1212/WNL.0000000000007527 |
N° Profil MNM : | 2019042 |
See also : |