Information About Gene Therapy
At the SADS Foundation, we are committed to providing information about the potential risks of gene therapy trials, and also connecting you to open studies. We are here for you as you make your decision about whether participating in a clinical trial for gene therapy is right for you.
FAQ’s
What is gene therapy?
Gene therapy offers the possibility of a permanent cure for conditions caused by a defect in a single gene – like SADS conditions.
How does gene therapy work?
Gene therapy works by replacing or inactivating disease-causing genes.
When will gene therapy clinical trials be available?
Clinical trials for gene therapy are getting closer, and may be available for certain conditions and genetic mutations in 2023.
What are the risks of being part of a clinical trial for gene therapy?
Gene therapy is still a relatively new therapy and we’re still learning about the possible risks. So far, some of the possible risks include unwanted immune system reaction, the virus used to deliver the therapy may target the wrong cells, infection, and possibly causing cancer. There may be other risks.
How do I decide whether to participate in a trial for gene therapy?
Become as educated as possible about gene therapy in general. Ask lots of questions about the trial itself-
- What are the risks and benefits?
- What safety protocols are in place?
- How long will I need to be monitored and where will that monitoring take place?
- Will I need to travel to be a part of the trial?
- Has this treatment been tested before?
- How long will the trial take?
- Have there been other trials similar to this one? If yes, what were the results?
- What kinds of patients will be in the clinical trial?
- How many patients are needed for the clinical trial?
- Will I find out about the results of the clinical trial?
Ultimately, the decision of whether to participate in a clinical trial for gene therapy is a personal decision, taking into account the particular trial and the risks and potential benefits of the trial.
If I start enrolling in a trial but change my mind, what happens?
Just because you start the process of enrolling in a clinical trial, you don’t have to continue if you think the risks are unacceptable, or the trial would be too hard on you or your family.
Where can I get more information about gene therapy trials?
At the SADS Foundation, we are committed to providing information about the potential risks of gene therapy trials, and also connecting you to open studies. We are here for you as you make your decision about whether participating in a clinical trial for gene therapy is right for you.
Resources
Questions? Reach Out To Us!
Please reach out to genevie@sads.org with any questions