The buildup of Gb3 in the cells can cause serious damage to vital organs, including the kidney, heart, nerves, eyes, gut and skin. Symptoms of Fabry disease can include decreased or absent sweat ...

A "productive" meeting with the FDA has given the firm confidence in its regulatory filing and commercialization plans for ST-920.

its wholly owned gene therapy product candidate for the treatment of Fabry disease. All dosed patients in the Phase 1/2 STAAR study evaluating isaralgagene civaparvovec have now completed at least ...