Title: | Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial |
Journal : | The Lancet. Neurology |
Authors: | Masson R ; Mazurkiewicz Beldzinska M ; Rose K ; Servais L ; Xiong H ; Zanoteli E ; Baranello G ; Bruno C ; Day JW ; Deconinck N ; Klein A ; Mercuri E ; Vlodavets D ; Wang Y ; Dodman A ; El-Khairi M ; Gorni K ; Jaber B ; Kletzl H ; Gaki E ; Fontoura P ; Darras BT |
Material Type: | Article |
Publisher: | England, 13/10/2022 |
General note: | Institut de Myologie |
Languages: | English |
Keywords : | biological markers ; children ; clinical trials ; motor skills ; proximal spinal muscular atrophy type 1 ; risdiplam ; SMN1-related proximal spinal muscular atrophy ; spinal muscular atrophy |
Pubmed / DOI : | Pubmed : 36244364 / DOI : 10.1016/S1474-4422(22)00339-8 |
N° Profil MNM : | 2022101 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/36244364 |
See also : |