Trusted Resources: Education
Scientific literature and patient education texts
Comparison of Nusinersen and Evrysdi in the Treatment of Spinal Muscular Atrophy
source: E3S Web of Conferences
year: 2021
authors: Xiaoying Zhu
summary/abstract: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.
organization: Healthcare decision and analysis, USA
DOI: 10.1051/e3sconf/202127103035
read more
Related Content
-
Neurofilament Levels in CSF and Serum in an Adult SMA Cohort Treated With NusinersenObjective: To retrospectively evaluate t...
-
Assessment of Cerebral Spinal Fluid Biomarkers and microRNA-Mediated Disease Mechanisms in Spinal Muscular Atrophy P...Cerebral spinal fluid (CSF) is a promisi...
-
Biogen to Launch Study of Higher Spinraza Dose in Patients With Later Onset SMABiogen announced it intends to initiate ...
-
Nusinersen for Spinal Muscular Atrophy in the United States: Findings From a Retrospective Claims Database AnalysisSpinal muscular atrophy (SMA) is a rare,...
-
Pediatric Spinal Muscular AtrophyWhat is spinal muscular atrophy (SMA)?Sp...
-
Lunch & Learn: Gene Therapy for SMAhttps://www.youtube.com/watch?v=YFEMR1ZH...
-
Safety and Efficacy Data Published for Novel Nusinersen Drug Delivery Method for Spinal Muscular Atrophy PatientsA recently published paper details the s...