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% ...

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

In a video posted to her Facebook page, the Conservative health critic said she was shocked to learn how badly B.C. ranks ...

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 ...

Rare Powassan virus case in Maine highlights serious tick-borne illness with no vaccine or treatment, causing neurological ...

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 ...

Experts say the system of EDRDs incentivizes companies to manufacture these drugs due to the high prices that can be charged ...