Type I SMA "new natural history": long-term data in nusinersen-treated patients.

ABSTRACT:

Objective

The aim of this paper was to report the 2-year follow-up in type I patients treated with Nusinersen and to assess whether possible changes in motor function are related to the subtype, age, or SMN2 copy number.

Methods

Sixty-eight patients, with ages ranging from 0.20 to 15.92 years (mean: 3.96; standard deviation: +3.90) were enrolled in the study. All patients were assessed using the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) and the developmental section of the Hammersmith Infant Neurological Examination (HINE-2) at the time they started treatment and 12 and 24 months after that.

Results

For both CHOP and HINE-2 repeated measures analysis of variance showed a significant difference (P < 0.001) between baseline

SUBMITTER: Pane M 

PROVIDER: S-EPMC7951096 | biostudies-literature | 2021 Mar

REPOSITORIES: biostudies-literature