Dror Bashan, President and CEO of Protalix Biotherapeutics talks about the company’s Fabry disease pipeline, that includes pegunigalsidase alfa, a plant based enzyme replacement therapy, that is currently under investigation in numerous clinical trials to assess its efficacy and safety in drug naïve patients as well as those previously treated with other enzyme replacement therapy (Replagal).
Fabry disease is an inherited lysosomal storage disorder that results from the buildup of a particular type of globotriaosylceramide or GL-3 in cells. Fabry disease affects many parts of the body. Signs and symptoms may include episodes of pain, particularly in the hands and feet (acroparesthesias); clusters of small, dark red spots on the skin called angiokeratomas; a decreased ability to sweat (hypohidrosis); cloudiness of the front part of the eye (corneal opacity); and hearing loss. Potentially severe complications can include progressive kidney damage, heart attack, and stroke.
To learn more about this and other lysosomal storage diseases, visit https://checkrare.com/diseases/lysosomal-storage-disorders/
