Fabry disease


When should we begin enzyme replacement therapy for our children with Fabry Disease?

The timing of initiation for enzyme replacement therapy (ERT) can vary depending on the wishes of the family, symptoms of the child, and the opinions of the doctor. Often, the treating physician will closely monitor gastrointestinal, kidney, and heart functions while also asking questions about the child's energy and pain. They often recommend beginning ERT when symptoms of the disease emerge and begin to affect daily life and when symptoms begin to progress. The parents wishes are always taken into account when determining the time to begin therapy. At this time, therapy rarely is started before 3 years of age. Please talk to your Fabry doctor or genetic counselor to come up with the best treatment and monitoring plan.

Mehta, A. (2017, January 5). Fabry Disease. Retrieved November 22, 2019, from http://www.ncbi.nlm.nih.gov/books/NBK1292/.

National Fabry Disease Foundation. (n.d.). Retrieved from http://www.fabrydisease.org.

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