| Issue |
E3S Web Conf.
Volume 271, 2021
2021 2nd International Academic Conference on Energy Conservation, Environmental Protection and Energy Science (ICEPE 2021)
|
|
|---|---|---|
| Article Number | 03035 | |
| Number of page(s) | 4 | |
| Section | Research on Energy Chemistry and Chemical Simulation Performance | |
| DOI | https://doi.org/10.1051/e3sconf/202127103035 | |
| Published online | 15 June 2021 | |
Comparison of Nusinersen and Evrysdi in the Treatment of Spinal Muscular Atrophy
USC Master graduate, School of Pharmacy, Healthcare decision and analysis, USA
* Corresponding author: xzhu4711@usc.edu
Spinal Muscular Atrophy (SMA) is a genetic neuromuscular disease that commonly affects children, and usually worsens with age that often leads to permanent disability and death for many of the SMA patients. Recently, two drugs are developed to improving the quality of life of SMA sufferers: Evrysdi and Nusinersen. This study is identified by a systematic literature review to compare two treatments. The comparison attempts to focus on mechanism, administration and clinical trials. The trials include the ENDEAR study for Nusinersen, and the FIREFISH study for Evrysdi. Due to the different baselines of two trials, matching-adjusted indirect comparison (MAIC) is used to “weighted” baseline characteristics to match each other across all the studies. Each of the trials highlighted the effectiveness for comparison. Both Nusinersen and Evrysdi have had a major and positive impact on improving the quality of life of SMA, and both therapies have been shown to be highly effective. Moreover, the indirect comparison with Matching Adjustment Indirect Comparison shows that Risdiplam is more effective as compared to Nusinersen. Nonetheless, the comparison is still inaccurate due to lack of real-world evidence from patients.
© The Authors, published by EDP Sciences, 2021
This is an Open Access article distributed under the terms of the Creative Commons Attribution License 4.0, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
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