Fabry disease
Clinical Research/Studies
Where do I learn more about clinical trials for Fabry disease?
The easiest way to learn more about studies and clinical trials in Fabry disease is to talk to a genetic counselor, a doctor that specializes in Fabry disease, or medical geneticist that is familiar with Fabry disease. However, studies focusing on treatments and cures are also posted at clinicaltrials.gov where you can search for "Fabry disease." It may help to avoid limiting your search to your state or country, as many studies will pay for your travel if you are eligible to join.
Another great resource for information on studies are the Fabry support groups. The support websites that may be of help are: The National Fabry Disease Foundation, Fabry Support and Information Group or FSIG
Individuals interesting in learning more about what it means to participate in a clinical trial may wish to visit webpages such as the Emory Genetic Clinical Trials Center Clinical Trials Resources Page to learn more about being a part of these studies.
Antidote also offers a handy tool to find clinical trials.
References
- National Fabry Disease Foundation. (n.d.). Retrieved from http://www.fabrydisease.org.
- Fabry Support & Information Group. (n.d.). Retrieved November 22, 2019, from http://www.fabry.org.
- Discover Fabry. (n.d.). Retrieved November 22, 2019, from https://www.discoverfabry.com/.
- Fabry Disease. (n.d.). Retrieved November 22, 2019, from http://rarediseases.org/rare-diseases/fabry-disease/.
- U.S. National Library of Medicine - clinicaltrials.gov. (n.d.). Retrieved from https://clinicaltrials.gov/.
- Mehta, A. (2017, January 5). Fabry Disease. Retrieved November 22, 2019, from http://www.ncbi.nlm.nih.gov/books/NBK1292/.
More Clinical Research/Studies Content
How do I find gene therapy studies for Fabry disease?
How many companies are conducting gene therapy studies in Fabry disease?
How do I find gene therapy studies for Fabry disease?
Gene therapy studies in Fabry disease are open to enrolling people with Fabry disease in the United States, Canada, Australia, and other countries. Most of the studies are focused on men with classic Fabry disease at this time, but in future studies women with classic Fabry disease and individuals living with non-classic Fabry disease will be included as well. There are several companies (4DMT, Freeline, Sangamo) exploring different methods of doing gene therapy in Fabry disease, so it is important to discuss the risks and benefits with a doctor familiar with the options to see which appears to make the most sense for a specific individual.
The easiest way to learn if you are candidate for a gene therapy trial in Fabry disease is to talk to healthcare provider that specializes in Fabry disease. However, studies focusing on gene therapy are also posted at clinicaltrials.gov where you can search for "Gene Therapy Fabry disease." It may help to avoid limiting your search to your state or country, as many studies will pay for your travel if you are eligible to join.
References
- Gene Therapy for Fabry disease. Fabry Disease News. https://fabrydiseasenews.com/gene-therapy-for-fabry-disease/ Accessed 12DEC2019
How many companies are conducting gene therapy studies in Fabry disease?
Gene therapy studies in Fabry disease are open to enrolling people with Fabry disease in the United States, Canada, Australia, and other countries. Most of the studies are focused on men with classic Fabry disease at this time, but in future studies women with classic Fabry disease and individuals living with non-classic Fabry disease will be included as well. There are several companies (4DMT, Freeline, Sangamo) exploring different methods of doing gene therapy in Fabry disease, so it is important to discuss the risks and benefits with a doctor familiar with the options to see which appears to make the most sense for a specific individual.
The easiest way to learn if you are candidate for a gene therapy trial in Fabry disease is to talk to healthcare provider that specializes in Fabry disease. However, studies focusing on gene therapy are also posted at clinicaltrials.gov where you can search for "Gene Therapy Fabry disease." It may help to avoid limiting your search to your state or country, as many studies will pay for your travel if you are eligible to join.
References
- Gene Therapy for Fabry disease. Fabry Disease News. https://fabrydiseasenews.com/gene-therapy-for-fabry-disease/ Accessed 12DEC2019
