Medicalive blog

Posts Tagged ‘genetic disorders’

WASHINGTON — The FDA has formally approved the enzyme replacement therapy velaglucerase alfa (VPRIV) — already okayed for pre-approval marketing — for pediatric and adult patients with Gaucher disease.  Gaucher disease is a rare genetic disorder affecting between 1 in 50,000 and 1 in 100,000 in the general population.

It limits production of the enzyme glucocerebrosidase, the lack of which allows fatty lipids to build up in the liver, spleen, bones, bone marrow, and nervous system.  Read the rest of this entry »