
Dr Robert Hopkin on Future Considerations for Management of Fabry Disease
Robert J. Hopkin, MD, clinical geneticist, Cincinnati Children's Hospital Medical Center, provides an overview of the evolving treatment landscape in Fabry disease.
With no treatment options for
Transcript
What are some future considerations regarding management of Fabry disease?
Fabry disease is a changing field constantly, and right now most of the patients who are being treated have very little enzyme production from the copy of the gene that's abnormal. So, in men, they only have 1 copy, and in women, they have 2 copies, but the cells with
We need to pay attention to the treatment options. We've gone from no treatment 20 years ago to now we have 2 treatments, and there are probably at least 10 groups studying newer options to improve the outcome for people with Fabry disease.
So, scientifically, it's an exciting time. Probably in the next 5 to 10 years, we will have some treatments that are less of a burden on the patients. And I think that it behooves the medical care providers and the patients with Fabry disease to keep an eye on this evolution so that we can take advantage of what we're learning as the science advances.
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