Fabry disease and treatment with agalsidase alpha: unsuspected cardiac arrhythmia in two heterozygous women. In reference to pharmacovigilance.

CASE REPORTS: Two women treated with agalsidase alpha for Fabry disease developed severe heart dysfunctions a few months after the beginning of enzyme replacement therapy (ERT). An adverse effect caused by the treatment was suspected; therefore we informed the French pharmacovigilance authorities about these two events.

CONCLUSIONS

Collecting data systematically about adverse effects which might be caused by drugs is essential for orphan diseases in order to detect potential adverse effects which cannot be suspected at first.