Experimental Treatments

Agalsidase alfa is an enzyme replacement therapy (ERT) designed to provide a functional alpha-galactosidase A (Gal A) enzyme to people with Fabry disease, which is expected to ease symptoms and slow disease progression. Agalsidase alfa is a concentrated, lab-made form of Gal A designed to temporarily provide patients with the enzyme they need to break down Gb3 and other molecules.

Gene therapy for Fabry disease is designed to introduce a healthy copy of the GLA gene inside cells to restore normal levels of the alpha-galactosidase A enzyme. Some potential gene therapies are now being investigated for the treatment of Fabry disease.

Substrate reduction therapy is an investigational treatment method for Fabry disease and other lysosomal storage disorders. Substrate reduction therapy takes its name from the fact that it reduces the amounts of the substrate of a certain enzyme. In this way, the therapy ensures that lack of this enzyme in Fabry disease is no longer a problem.