Some of the biggest U.S. investment announcements from Europe so far have come from the pharmaceutical sector, with Swiss ...

On Saturday, May 24, the 78th World Health Assembly (WHA) voted to adopt a resolution to establish a global framework to ...

Wilson's new company, Philadelphia-based Gemma Biotherapeutics, plans to start testing gene therapies in Brazil this year.

A new rapid blood test for newborns could potentially detect genetic mutations linked to thousands of rare diseases all at ...

Many rare disease patients do not receive a formal genetic diagnosis, but a European data-sharing project aimed to give them ...

This is a historic moment in decades-long efforts to appropriately address the needs of the hundreds of millions worldwide ...

Corina and Destiny lost their first child, Ahi o Te Aroha, at six months old. Although genetic testing identified a variant ...

In addition to sickle cell disease, rilzabrutinib has received orphan drug designation for immune thrombocytopenia (ITP) in the US, the EU, and Japan, for warm autoimmune hemolytic anemia (wAIHA) in ...

A single, untargeted proteomics test for rare genetic diseases can analyze thousands of proteins from a 1ml sample of blood.

A groundbreaking blood test promises to transform the diagnosis of rare genetic diseases in babies and children, offering ...

Stocks slipped in Asia on Friday and the U.S. dollar drooped with Treasury yields as investors digested an appeals court kept ...

Five families with children who have the newly discovered rare condition, ReNU syndrome, recently gathered in a D.C. park.