Information About Gene Therapy
Gene therapy and SADS conditions
Gene therapy presents exciting potential therapy for conditions that result from genetic mutations, including SADS conditions. Gene therapy aims to cure or stop the progression of a disease by fixing a faulty gene or by replacing that gene with a healthy one.
Developing gene therapy for SADS conditions
Gene therapies are not yet commercially available for SADS conditions, though some are in clinical trials. We are determined to have the voice of SADS patients at the center of all research and we work with companies and researchers to ensure that link is always present.
We will continue to keep SADS families informed of new research and clinical trials. If you are not yet signed up to receive notifications and other news, you can do that here.
Current clinical trials for gene therapies related to SADS conditions
There are currently three gene therapy clinical trials enrolling patients with PKP2 variants that cause Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC), also known as Arrhythmogenic Cardiomyopathy (ACM). There are also gene therapies being developed and studied in the lab in animal models for Long QT Syndrome and CPVT.
Understanding clinical trials for gene therapies
Clinical trials are a key step in advancing gene therapies, and SADS families may be qualified to participate in this research. Choosing whether or not to participate is a personal decision without a “right” or “wrong” answer. There are many factors to consider when weighing if it makes sense for you or a family member to participate and the team here at SADS is committed to ensuring you have everything you need to make an informed decision.
Frequently asked questions
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What is gene therapy and how does it work
Gene therapy presents exciting potential therapy for conditions that result from genetic mutations, including SADS conditions. Gene therapy aims to cure or stop the progression of a disease by fixing a faulty gene or by replacing that gene with a healthy one.
When will gene therapy clinical trials be available?
Gene therapies are not yet commercially available for SADS conditions, though some are in clinical trials. We are determined to have the voice of SADS patients at the center of all research and we work with companies and researchers to ensure that link is always present.
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, and risk of infection. There may be other risks that are not yet known.
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.
Additional Resources
History of Gene Therapy
Questions? Reach Out To Us!
Please reach out to genevie@sads.org with any questions
Learn more from leading SADS experts
Choosing to participate in a study is an important personal decision. Before you participate in a study, know the risks and potential benefits and discuss all options with your health care provider and other trusted advisors.
SADS Foundation lists current opportunities to participate in studies that we have been informed of. Information on SADS.org is provided by study sponsors and investigators, and they are responsible for ensuring that the studies follow all applicable laws and regulations. Please note that some partners have paid to have their trials posted. This does not imply recommendation or favor of the SADS Foundation, as we do not endorse any particular study.