Experimental Treatments

Agalsidase alfa is an enzyme replacement therapy (ERT) designed to provide a functional alpha-galactosidase A enzyme to people with Fabry disease, which is expected to ease symptoms and slow disease progression.

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. Gene editing therapy is a slightly different approach and involves making precise modifications to specific segments of a person’s own DNA.

Substrate reduction therapy works to reduce the toxic accumulation of certain fatty molecules associated with Fabry disease through a mechanism that is independent of the alpha-galactosidase A enzyme.