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Table 4 Current proposed guidelines for starting enzyme replacement therapy in Fabry disease patients

From: Fabry disease

Subpopulation

Guidelines for onset of ERT

Adult males (over 16 years)

At time of diagnosis of Fabry disease

Boys

At time of development of significant symptoms or if asymptomatic, consider at 7-10 years

Females (all ages)

Symptoms or evidence of progression of organ involvement

  1. Note: the recommendations are proposed guidelines from an international panel of physicians with expertise in Fabry disease [51]. They are currently not followed in every country and may be revised when additional studies on the outcome of the safety and efficacy of enzyme replacement therapy become available.
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172