Nusinersen in pediatric and adult patients with type III spinal muscular atrophy.

ABSTRACT:

Objective

We report longitudinal data from 144 type III SMA pediatric and adult patients treated with nusinersen as part of an international effort.

Methods

Patients were assessed using Hammersmith Functional Motor Scale Expanded (HFMSE), Revised Upper Limb Module (RULM), and 6-Minute Walk Test (6MWT) with a mean follow-up of 1.83 years after nusinersen treatment.

Results

Over 75% of the 144 patients had a 12-month follow-up. There was an increase in the mean scores from baseline to 12 months on both HFMSE (1.18 points, p = 0.004) and RULM scores (0.58 points, p = 0.014) but not on the 6MWT (mean difference = 6.65 m, p = 0.33). When the 12-month HFMSE changes in the treated cohort were compared to an external cohort of untreated patients, in all untreated patients old

SUBMITTER: Pera MC 

PROVIDER: S-EPMC8351459 | biostudies-literature | 2021 Aug

REPOSITORIES: biostudies-literature