Fabry disease

Clinical Research/Studies

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. In 2019, there are more than four companies ( 4DMT, AvroBio, 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.

SOURCE: Emory University - Department of Human Genetics in collaboration with ThinkGenetic • https://www.thinkgenetic.com/diseases/fabry-disease/clinical-research/71359 • DATE UPDATED: 2019-12-12

References

Gene Therapy for Fabry disease. Fabry Disease News. https://fabrydiseasenews.com/gene-therapy-for-fabry-disease/ Accessed 12DEC2019

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