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 ...

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

The pharmaceutical industry has a fresh sense of direction after President Donald Trump’s tax-and-spending law included a ...

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 ...

Rising high school senior Khartik Uppalapati says he felt alienated when he was diagnosed with a rare lymphatic disorder ...

MrBeast’s new CEO is hitting Wall Street as YouTuber Jimmy Donaldson’s media empire looks to develop long-term brand ...