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. 2021 Mar;63(3):311-319.
doi: 10.1002/mus.27116. Epub 2020 Dec 19.

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

Er Chen  1 , Stacy Dixon  2 , Rupali Naik  3 , Josh M Noone  4 , J Daniel Buchenberger  4 , Sarah M Whitmire  4 , Rosalina Mills  4 , William Arnold  5
Affiliations

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

Er Chen et al. Muscle Nerve. 2021 Mar.

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.

Conclusions: Despite therapeutic advances, there remain significant unmet needs for SMA. Challenges with administration and barriers to access potentially limit the number of patients treated or delay treatment. Continued efforts are needed to develop more treatment options and to improve access to treatments.

Keywords: SMA; burden; nusinersen; patient reported outcome; spinal muscular atrophy; unmet need.

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Conflict of interest statement

Study funded by Genentech Inc., A member of the Roche Group. Er Chen is an employee of GNE. Rupali Naik is a contracting employee of GNE. Josh M Noone, Daniel Buchenberger, Sarah M Whitmire and Rosalina Mills received research funding from GNE for the design and conduct of the research and writing of the paper. Stacy Dixon was contracted with GNE as clinical advisor of this project, no financial compensation was provided. Previously served as a paid member for an advisory board for Genentech. Additionally, have served as a paid member on several advisory boards for Biogen. William Arnold was contracted with GNE as clinical advisor of this project, no financial compensation was provided. Previously served as a consultant to Genentech and F. Hoffmann‐La Roche AG.

Figures

FIGURE 1
FIGURE 1
Adult and pediatric patient satisfaction with nusinersen (n = 77) [Color figure can be viewed at wileyonlinelibrary.com]
FIGURE 2
FIGURE 2
Patient/caregiver reported reasons for not receiving nusinersen (n = 24) [Color figure can be viewed at wileyonlinelibrary.com]
FIGURE 3
FIGURE 3
A, Time to insurance approval by SMA type. B, Proportion of patients with SMA treated by time of treatment initiation and age (n = 77) [Color figure can be viewed at wileyonlinelibrary.com]
See this image and copyright information in PMC

References

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