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.
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.
Authors: C M Eng; N Guffon; W R Wilcox; D P Germain; P Lee; S Waldek; L Caplan; G E Linthorst; R J Desnick Journal: N Engl J Med Date: 2001-07-05 Impact factor: 91.245
Authors: Raphael Schiffmann; Markus Ries; Margaret Timmons; John T Flaherty; Roscoe O Brady Journal: Nephrol Dial Transplant Date: 2005-10-04 Impact factor: 5.992
Authors: M Beck; R Ricci; U Widmer; F Dehout; A García de Lorenzo; C Kampmann; A Linhart; G Sunder-Plassmann; G Houge; U Ramaswami; A Gal; A Mehta Journal: Eur J Clin Invest Date: 2004-12 Impact factor: 4.686
Authors: Ales Linhart; Christoph Kampmann; José L Zamorano; Gere Sunder-Plassmann; Michael Beck; Atul Mehta; Perry M Elliott Journal: Eur Heart J Date: 2007-05-05 Impact factor: 29.983