Title: | Two-year efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA) |
Journal : | Journal of neurology |
Authors: | Oskoui M ; Day JW ; Deconinck N ; Mazzone ES ; Nascimento A ; Saito K ; Vuillerot C ; Baranello G ; Goemans N ; Kirschner J ; Kostera-Pruszczyk A ; Servais L ; Papp G ; Gorni K ; Kletzl H ; Martin C ; McIver T ; Scalco RS ; Staunton H ; Yeung WY ; Fontoura P ; Mercuri E |
Material Type: | Article |
Publisher: | Germany, 03/02/2023 |
Languages: | English |
Keywords : | clinical trials ; proximal spinal muscular atrophy type 3 ; risdiplam ; SMN1-related proximal spinal muscular atrophy ; spinal muscular atrophy |
Lien associé : |
Texte complet disponible sur PubMed Central® (PMC) Texte complet disponible sur PubMed Central (PMC) |
Pubmed / DOI : | Pubmed : 36735057 / DOI : 10.1007/s00415-023-11560-1 / Pubmed : 37071150 / DOI : 10.1007/s00415-023-11658-6 |
N° Profil MNM : | 2023021 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/36735057 |