Motor function in type 2 and 3 SMA patients treated with Nusinersen: a critical review and meta-analysis.

ABSTRACT:

Background

There is an increasing number of papers reporting the real world use of Nusinersen in different cohorts of SMA patients.

Main body

The aim of this paper was to critically review the literature reporting real world data on motor function in type 2 and 3 patients treated with Nusinersen, subdividing the results according to SMA type, age and type of assessment and performing a meta-analysis of the available results. We also report the available data collected in untreated patients using the same measures. Of the 400 papers identified searching for Nusinersen and spinal muscular atrophy, 19 reported motor function in types 2 and 3: 13 in adults, 4 in children and 2 included both. Twelve papers reported untreated patients' data. All studies reported positive changes o

SUBMITTER: Coratti G 

PROVIDER: S-EPMC8515709 | biostudies-literature | 2021 Oct

REPOSITORIES: biostudies-literature