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Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B | NEJM
Source: nejm.org
Article summary:
1. This article discusses the use of gene therapy with etranacogene dezaparvovec for treating moderate-to-severe hemophilia B.
2. The study found that this gene therapy was superior to prophylactic factor IX in terms of reducing the annualized bleeding rate, and had a favorable safety profile.
3. Benefits and safety were observed in participants with predose AAV5 neutralizing antibody titers of less than 700.
Article analysis:
This article is a research summary from the New England Journal of Medicine (NEJM) discussing the use of gene therapy with etranacogene dezaparvovec for treating moderate-to-severe hemophilia B. The study found that this gene therapy was superior to prophylactic factor IX in terms of reducing the annualized bleeding rate, and had a favorable safety profile.
The article appears to be reliable and trustworthy as it is published by a reputable journal, NEJM, which has stringent standards for publishing research articles. Furthermore, the authors are well qualified experts in their respective fields, providing credibility to the findings presented in the article. Additionally, there is full disclosure regarding funding sources and potential conflicts of interest at the end of the article, which further adds to its trustworthiness.
However, there are some potential biases that should be noted when considering this article's trustworthiness and reliability. Firstly, it is funded by uniQure and CSL Behring who both have an interest in promoting their product (etranacogene dezaparvovec). Secondly, while there is full disclosure regarding funding sources and potential conflicts of interest at the end of the article, it does not provide any information about how these funds were used or if they influenced any decisions made during the course of conducting or reporting on this study. Finally, while benefits and safety were observed in participants with predose AAV5 neutralizing antibody titers of less than 700, it does not provide any information about how these results may differ for those with higher titers or other factors such as age or gender that could influence outcomes.