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.