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 ...

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

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

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 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 ...

For many rare disease patients and their families, this often results in years of uncertainty, misdiagnosis, and limited or ...

Vinay Prasad, a top official at the Food and Drug Administration, has suddenly departed after a series of controversial ...

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

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

With the initiative, families can have their baby’s full genetic code sequenced at no cost to identify potentially serious ...