Orphan
Orphan Companies (3)
Orphan News
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News First treatment for Acid Sphingomyelinase Deficiency approved by US FDA
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. -
News Rare diseases care pathway: a major challenge to harmonize patient management
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...
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