Our inbox has been flooded with emails from viewers and readers who want to share their personal stories — people who want to ...

The study will compare NHS and social data to track long term impact of childhood blood disorders.

PharmaEssentia will spend $46 million to build a new manufacturing facility in Puerto Rico. The move forms part of the ...

This Rare Disease Day, observed on Feb. 28 worldwide, the American Kidney Fund is committed to improving the understanding of ...

Are autoimmune diseases fatal? Normally, your immune system fights off disease and protects your body from infection. However, when you have an autoimmune disease, your immune system ...

The clots occurred in two to three out of every 100,000 people who received the vaccine.

Seventy-five percent of patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN) who were treated with the antibody-drug conjugate (ADC) pivekimab sunirine (PVEK) had a complete response, ...

PPT1 is a recombinant protein used to treat an ultra-rare disease called Batten disease CLN1 for which there is ...

James Griffin, diagnosed with sickle cell at age two, shares how blood donations enabled his transformation from frequent hospital patient to medical professional and advocate.

Further testing found the child had pneumonia and extremely high liver enzymes. She was sent home with antibiotics and referred to a gastrointestinal specialist. That was the start of a years-long ...

Progressive multifocal leukoencephalopathy (PML) is a rare but serious brain infection. It gradually destroys brain tissue ...

The Orphan Drug Act revolutionized rare disease treatment, yet rising costs and access issues challenge equitable health care solutions.