D P Germain, C Boucly, R Y Carlier, E Caudron, P Charlier, F Colas, F Jabbour, V Martinez, S Mokhtari, D Orlikowski, N Pellegrini, C Perronne, H Prigent, R Rubinsztajn, K Benistan
Extraction and purification of an acid β-glucosidase from human placenta (alglucerase) for the treatment of Gaucher disease, replaced a few years later by a recombinant enzyme (imiglucérase, Cerezyme(®)), has paved the way to the development of enzyme replacement therapies (ERT) for the treatment of lysosomal storage diseases (LSD) among which Fabry disease for which the long-term efficacy of the two currently available preparations (agalsidase alfa, Replagal(®) and Fabrazyme(®)) is still being investigated...
December 2010: La Revue de Médecine Interne