Nusinersen for children with type I spinal muscular atrophy: 4 years' clinical experience in Turkish cohort.

ABSTRACT:

Background

SMA Type 1 is the most severe form of spinal muscular atrophy with early symptom onset, limited motor development, and poor prognosis. Recent genetic-based therapies, such as nusinersen, have transformed disease outcomes. We aimed to evaluate the long-term effects of nusinersen on motor, bulbar, and respiratory functions in both symptomatic and presymptomatic SMA Type 1 patients over a period of up to 4 years.

Methods

This prospective, non-interventional study included 310 patients with genetically confirmed spinal muscular atrophy at 24 pediatric neurology centers in Turkey. Patients treated with nusinersen were divided into five age-based cohorts at treatment initiation: Cohort A (0-3 months), Cohort B (4-6 months), Cohort C (7-12 months), Cohort D (13-24 months

SUBMITTER: Bektas O 

PROVIDER: S-EPMC11983886 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature