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 ...

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 ...

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 head of Italian group Recordati expects treatments for rare diseases to be exempted from the 15% tariff the United States plans to impose on imported European pharmaceutical products, he said on ...

Takeda reported a 30% rise in Q1 profit and beat EPS estimates, offsetting Vyvanse losses with growth in oncology, plasma, ...

For most Americans, the letters "NTM" don’t mean anything. But for tens of thousands of people across the country, they ...