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

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

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

The Senate Parliamentarian reversed course on a small provision in the One Big Beautiful Bill Act that could boost cures for rare diseases.

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 ...

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 ...

Patients with rare diseases are increasingly working to find and fund their own cures. But should they have to?

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

GREENSBORO, N.C. — More than 300 million people in the world live with a rare disease. More than 70% of rare diseases are genetic, and they may start in childhood. So, early diagnosis is crucial.

When every person can be treated personally, current rare disease patients won't be left out since every patient will have their own "rare disease" in a way. Every disease and every person matters.