Zoekresultaten - 2 results
A retrospective, non-interventional cohort study to compare adults with SMA receiving risdiplamwith untreated patients: Study design.
Publication Type: Scientific poster, presentation or proceeding Authors: Simpson, A.; Batech, M.; Carmona, A.; Lee, S.; Poll, A.; Edwards, S.; Guittari, C.; Sutherland, C.; Marini-Bettolo, C.; Walter, M.; Marlène Jagut; Haberlová, J.; Hodgkinson, V.; Yiu, ...
International collaboration to improve knowledge on myotonic dystrophy type 2
myotonic dystrophy type 2 (DM2), which aims to facilitate collaborative research and clinical trials. Objectives: This study aimed to assess DM2 patients included in the network, and to analyse their ...