the study is as many as 52 weeks, or an entire year. The secondary outcome measures the percent change in LDL-C compared to the pre-trial levels. Additionally, it will compare the percent change in lipid parameters also to pre-trial levels. Each of these tests will be done over 12 weeks. The final measurements in the trial are related to the number of adverse, product-related events. As well as once again measuring the percent change in lipid parameters compareto the baseline. Both of these measurements will be monitored for a period of 260 weeks. I think that there are many benefits to simply participating in these trials. For example, I hold a firm belief that a “risky” trial beats the hell out of suffering with a possibly curable disease. Or in other words, if there is even a slight chance of success, the benefits almost always
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outweigh the costs. I must admit that there are of course potential pitfalls in any medical trial, such as contracting a separate disease or somehow worsening your own condition. That being said, I still think that the trial is very promising for multiple reasons, the least of which being how many universities and medical centers are willing to undertake the trial. They have all put their confidence in these tests, so I believe that alone is nearly enough to capture my attention. The final evidence I would give to support the trial is how upstanding the information surrounding it is. It is all presented very clearly and effectively, and every aspect of the trial seems very trustworthy.
Atherosclerosis, Clinical trial, Familial hypercholesterolemia, Adverse event, Gene Therapy Study
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