Abstract
Fabry disease (FD) is a metabolic disorder with clinical onset during childhood or adolescence. A high percentage of patients with FD suffer from neuropathic pain and gastrointestinal (GI) complaints. The positive effect of available treatments (enzyme replacement therapy with agalsidase alfa/beta (ERT) and migalastat) on these symptoms can improve patients’ quality of life. We investigated the minimum time of follow-up in FD to detect the statistically significant impact on neuropathic pain and GI complaints after treatment with available therapies.
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