"Sickle cell disease is a devasting disorder that affects approximately 100,000 people in the U.S., leading to anemia, severe ...

In the world of international child development and orphan care, it's not uncommon for children with families to declare ...

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 ...

Thomas Ho, 76, Vietnamese orphan, speaks about his journey in 1975, to the U.S. Ho and other orphans will be back Vietnam to ...

The government is expected to launch a new inquiry into the pricing and reimbursement system of pharmaceuticals in Sweden, ...

Beam Therapeutics Inc. (NASDAQ:BEAM) has secured U.S. FDA orphan drug designation for BEAM-302, its pioneering genetic medicine targeting alpha-1 antitrypsin deficiency (AATD), a rare inherited ...