Treatment of Fabry disease

PubMed, the Internet portal of biomedical and life sciences literature, indexed an interesting article, entitled ”Clinical development of agalsidase-beta for the treatment of Fabry disease” (Med Sci (Paris) 2005 Dec;21(11):57-61). Author is Germain DP from Unite de Genetique Clinique, Hopital Europeen Georges Pompidou, Paris, France. Agalsidase beta has been developed for enzyme replacement for the treatment of Fabry disease, a rare and debilitating genetic disease caused by deficient activity of the lysosomal enzyme alpha-galactosidase A. Lack of this enzyme leads to a progressive tissular accumulation of globotriaosylceramide (Gb3), resulting in life-threatening renal, cardiac and cerebrovascular complications. Agalsidase beta (Fabrazyme(R)), was approved by the European Agency for the Evaluation of Medicinal Products (EMEA) in 2001. To access the full abstract of the article, click here.