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Nephrol Dial Transplant (2003) 18: 33-35
© 2003 European Renal Association-European Dialysis and Transplant Association

Rapid Communication

Enzyme replacement therapy in Anderson–Fabry's disease: beneficial clinical effect on vital organ function

Gert De Schoenmakere, Dominique Chauveau and Jean-Pierre Grünfeld

Service de Néphrologie, Hôpital Necker, Paris, France

Abstract

Two recent randomized trials pointed out the beneficial effect of enzyme replacement therapy on biochemical parameters in patients with Anderson–Fabry's disease. Clinical end-points, such as amelioration or stabilization of renal function deterioration, or improvement of left ventricular hypertrophy have not been evaluated in depth. We report the case of a patient whose moderately impaired renal function was stabilized with the start of enzyme treatment. In addition, left ventricular hypertrophy tended to regress. To our knowledge this is the first observation of clinical efficacy of the enzyme replacement therapy in Anderson–Fabry's disease in patients with moderately impaired renal function.

Keywords: Anderson–Fabry's disease; beneficial clinical effect; enzyme replacement therapy; vital organ function

Notes

Correspondence and offprint requests to: Jean-Pierre Grünfeld, Service de Néphrologie, Hôpital Necker, 149 rue de Sèvres, F-75743 Paris Cedex 15, France. Email: jean-pierre.grunfeld{at}nck.ap-hop-paris.fr


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