The Food and Drug Administration has approved Cerdelga (eliglustat) for the treatment of adult patients with the Type 1 form of Gaucher disease.
An experimental drug for treating a rare genetic disease appears effective, according to a late-stage clinical trial.
Drug makers could strike big with new treatments for rare diseases, according to a new report by a market research firm.
The Food and Drug Administration has approved a new treatment for a rare genetic disorder that affects 10,000 people worldwide.
British drug maker Shire is looking to make a biotech drug for a rare genetic disorder at a new manufacturing plant in Massachusetts.
Pfizer is partnering with San Diego biotech company Zacharon Pharmaceuticals to develop treatments for rare genetic disorders Zacharon said Thursday.
The Food and Drug Administration has declined to approve a biologic treatment for a rare genetic disorder by Israeli drug maker Protalix Biotherapeutics, Protalix said Friday.
Generic drug maker Hospira will fill and package several drugs for Genzyme, according to published...
Biotech company Genzyme will pay $175 million to the federal government as part of an...
The Food and Drug Administration has approved a new drug for treating a rare genetic...