Orphan

Enzyme replacement therapeutic Xenpozyme has received US FDA approval with an orphan drug designation to treat symptoms of Acid Sphingomyelinase Deficiency, a rare genetic disease.

In France, rare diseases are numerous: between 7,000 and 8,000 diseases are currently accounted for, and concern more than 3 million French people, accounting for almost 4,5% of the total population. The organization of screening and care for these ill...