Biogen raised its annual profit forecast and beat estimates for quarterly results on Thursday, as strong demand for its rare ...

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 ...

British mum Laura Mattinson was devastated to learn her one-year-old son Ezra had been diagnosed with Leigh syndrome after he ...

Chris Kirkpatrick is in awe that his former ‘NSync bandmate Justin Timberlake performed a full tour amid a battle with Lyme ...

Biogen Inc. (NASDAQ:BIIB) reported on Thursday that the second quarter of 2025 adjusted earnings per share were $5.47, up 4% ...

The primary safety risks associated with gene therapy include immune reactions and off-target effects in unintended organs, such as the liver, lungs, and spleen. Nevertheless, Ali and Townsend note ...

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 ...

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

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

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

Dr. Michael Wagner covers the basics of epithelioid hemangioendothelioma, a rare vascular cancer, spanning symptoms, ...