Therapy areas

Fabry is caused by a defective gene located on the X chromosome.

Gaucher is more common in people with Ashkenazi Jewish heritage.

MPS I has also been called Hurler, Hurler-Schele and Scheie Syndromes.

Women have about 2 to 3 times the risk for multiple sclerosis than men.

Pompe disease occurs in approximately 1 in 40,000 live births.