Early experiences of nusinersen for the treatment of spinal muscular atrophy: Results from a large survey of patients and caregivers.

ABSTRACT:

Background

This study aimed to examine the early experience of nusinersen for spinal muscular atrophy (SMA) from the patient and caregiver perspective.

Methods

A 54-item online survey was administered to adult patients and caregivers of pediatric patients diagnosed with SMA.

Results

Overall, respondents (56 patients and 45 caregivers) were satisfied with nusinersen. Satisfaction was highest on changes in energy, stamina, and motor function and lowest on treatment administration and overall time commitment. Differences were noted for treatment effect sustained over time as reported by adult patients vs caregivers reporting on behalf of pediatric patients. Respondents reported insurance approval as a key barrier to access, particularly among adult patients.

Conclusio

SUBMITTER: Chen E 

PROVIDER: S-EPMC7986200 | biostudies-literature | 2021 Mar

REPOSITORIES: biostudies-literature