Longitudinal Efficacy of Nusinersen Treatment on Health-Related Quality of Life and Independence in Children With Later-Onset Spinal Muscular Atrophy.

ABSTRACT:

Introduction/aims

The rising use of disease-modifying therapy is progressively impacting the health-related quality of life (HRQoL) of patients with spinal muscular atrophy (SMA) in their daily lives. This study aimed to evaluate the changes in HRQoL and independence in children with later-onset SMA receiving longitudinal treatment with nusinersen.

Methods

Forty-nine pediatric patients with later-onset SMA (symptom onset after 6 months of age) and their caregivers were enrolled. The HRQoL of patients evaluated by the proxy-reported Pediatric Quality of Life Inventory 3.0 Neuromuscular Module (PedsQL NMM) and the independence level determined by the SMA Independence Scale-Upper Limb Module (SMAIS-ULM) were assessed. Caregiver HRQoL was assessed using the Pediatric Quality of

SUBMITTER: Huang S 

PROVIDER: S-EPMC11799406 | biostudies-literature | 2025 Mar

REPOSITORIES: biostudies-literature