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Real-world Adherence to Nusinersen in Adults with Spinal Muscular Atrophy in the US: A Multi-site Chart Review Study
Lauren Elman1, Bora Youn2, Crystal M Proud3
1University of Pennsylvania, Philadelphia, PA, USA.
Journal of Neuromuscular Diseases
|August 29, 2022
Summary
Real-world adherence to nusinersen for spinal muscular atrophy (SMA) treatment is high. Most adults with SMA received their nusinersen doses on time, demonstrating good treatment compliance.
Area of Science:
- Neurology
- Rare Diseases
- Pharmacology
Background:
- Spinal muscular atrophy (SMA) is a rare genetic neuromuscular disorder.
- Nusinersen is an approved treatment for SMA, but real-world adherence data are limited.
- This study evaluated adherence to nusinersen in adult SMA patients.
Discussion:
- High adherence rates (92%) were observed in adult SMA patients initiating nusinersen.
- The majority of patients remained on nusinersen throughout the study period.
- Most patients who missed a dose resumed the treatment schedule, indicating good persistence.
Key Insights:
- Adult SMA patients demonstrate strong adherence to the nusinersen dosing schedule.
- Real-world data suggest nusinersen is well-tolerated and manageable in terms of adherence.
- Consistent dosing is crucial for optimal outcomes in SMA management.
Outlook:
- Further research should explore long-term adherence trends and factors influencing non-adherence.
- Understanding adherence patterns can inform strategies to optimize SMA treatment.
- Real-world evidence supports the feasibility of maintaining nusinersen therapy in adult SMA populations.
