Search results - 4 results
A retrospective, non-interventional cohort study to compare adults with SMA receiving risdiplamwith untreated patients: Study design.
Walter, M.; Marlène Jagut; Haberlová, J.; Hodgkinson, V.; Yiu, E.; Murphy, L.; TREAT-NMD Global Data systems Oversight Committee (TGDOC); TREAT-NMD Global Registry Network for SMA; Roe, T.; ...
Belgian neuromuscular diseases registry (BNMDR): Annual report 2017
which the four sub-types of Spinal Muscular Atrophy (SMA) are divided in four distinct disease groups. When pooled together, SMA was ranked 9th in the list. For two defined groups of diseases, i.e. DMD ...
Nusinersen treatment significantly improves hand grip strength, hand motor function and MRC sum scores in adult patients with spinal muscular atrophy types 3 and 4
Claeys Source: Journal of Neurology (2020) Keywords: SMA · Spinraza® · Muscle strength · ActivLim · Outcome measures · Natural history Abstract: Abstract Background Nusinersen recently became available as ...
Collaborative data collection by TREAT-NMD Registries to support post-marketing surveillance in Spinal Muscular Atrophy
Goemans; Anna Ambrosini; Craig Campbell Source: TREAT-NMD conference, TREAT-NMD, Leiden, The Netherlands (2019) Keywords: Data collection post-marketing surveillance SMA TREAT-NMD Abstract: Spinal muscular ...