Alpha-galactosidase A (AGAL): An enzyme that breaks down certain lipids in the body; its deficiency leads to Fabry disease. Lysosomal storage disorder: A group of inherited metabolic diseases that ...

Brian has Fabry disease, a lysosomal storage disorder that can cause a variety of symptoms. Start the day smarter. Get all the news you need in your inbox each morning. “It's a condition where ...

Interest in lysosomal storage diseases (LSDs ... enzyme-replacement therapies can duplicate its economic success. Fabry's disease is an X-linked disorder caused by a deficiency of α-galactosidase.

Sanofi is still running studies of the glycosphingolipid (GSL) inhibitor in rare lysosomal storage diseases like Gaucher disease type 3, Fabry disease and GM2 gangliosidosis (also known as Tay ...

Italian pharma Chiesi has begun a new rare diseases division as it builds ... Chiesi markets treatments for the lysosomal storage disorders (LSDs) alpha‑mannosidosis and nephropathic cystinosis ...