Biogen raised its annual profit forecast on Thursday, betting on strong demand for its rare disease drugs such as Skyclarys ...

Katherine Stueland, GeneDx CEO, joins 'Squawk Box' to discuss diagnosing children with rare diseases, cost structure of the ...

From symptoms to the illness' risk factors — here’s what you need to know about the disease, which can only be detected by ...

Longtime drug developer Suma Krishnan was 51 when she cofounded Krystal Biotech. Now the company has one gene therapy on the ...

(Reuters) -Biogen raised its annual profit forecast on Thursday, betting on strong demand for its rare disease drugs such as ...

Thousands of years of endogamy may have shaped genetic disease risk in India. Scientists are now trying to identify those ...

The survey primarily focused on rare disease patients between the ages of 16 and 23, as well as caregivers between 25 to 55 years old. Hemophilia, albinism, cystic fibrosis and Gaucher disease were ...

In 2023 the FDA published guidance on rare diseases. The guidance recognises that many rare diseases are serious conditions ...

Opinion: The problem that occupies much of the UBC Therapeutics Initiative’s time is how to evaluate the scientific evidence ...

A Fontana child is fighting for her life against a rare genetic disorder called Batten disease while her parents are trying ...

Severe flu seasons in recent years have brought to light a little-known danger of influenza infections in kids: a rare brain ...

The NDP may be on the verge of blowing up B.C.’s process for drug approvals after 10 experts resigned from the province’s ...