Fabry disease


Is there gene therapy for Fabry disease?

Fabry is believed to be a good condition to treat with gene therapy because it is caused by a single gene (GLA) and it doesn't take a large amount of working enzyme to make a difference in the medical issues. For gene therapy to work, scientists need figure out the best way to get the working gene into the right place in the DNA in as many cells as possible WITHOUT causing side effects like an allergic reaction.

Although not yet available to everyone, 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.

Fabry disease revisited: Management and treatment recommendations for adult patients. Ortiz A, Germain DP, Desnick RJ, Politei J, Mauer M, Burlina A, Eng C, Hopkin RJ, Laney D, Linhart A, Waldek S, Wallace E, Weidemann F, Wilcox WR. Mol Genet Metab. 2018 Apr;123(4):416-427. doi: 10.1016/j.ymgme.2018.02.014. Epub 2018 Feb 28. Review. https://www.sciencedirect.com/science/article/pii/S1096719217307680?via%3Dihub Retrieved 12DEC19.

Tan, Victoria, Gene Therapy for Fabry Disease, Fabry Disease News, https://fabrydiseasenews.com/gene-therapy-for-fabry-disease/ Accessed 12DEC19.

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