Nusinersen in adult patients with 5q spinal muscular atrophy: A multicenter observational cohorts' study.

ABSTRACT:

Background and purpose

The aim was to assess the safety and efficacy of nusinersen in adult 5q spinal muscular atrophy (SMA) patients.

Methods

Patients older than 15 years and followed for at least 6 months with one motor scale (Hammersmith Functional Motor Scale Expanded, HFMSE; Revised Upper Limb Module, RULM) in five referral centers were included. The clinical and patients' global impression of change (CGI-C and PGI-C) were recorded in treated patients at the last visit. Functional scales (Egen Klassification, EK2; Revised Amyotrophic Lateral Sclerosis Functional Rating Scale, ALSFRS-R) and the percentage predicted forced vital capacity were collected when available.

Results

Seventy-nine SMA patients (39 treated with nusinersen) were included. Compared with untrea

SUBMITTER: Vazquez-Costa JF 

PROVIDER: S-EPMC9796289 | biostudies-literature | 2022 Nov

REPOSITORIES: biostudies-literature