The mother of a woman with a rare genetic disorder fears she is "helpless" in watching her daughter's condition decline ...

Janus kinase (JAK) inhibitors look to be a good treatment option for the newly recognized, rare autoimmune disease known as ...

A new type of bone marrow transplant can cure sickle cell disease with only half of the donor's cell proteins matching, ...

COPA syndrome causes lung hemorrhaging starting in childhood. But one in three people with the disease mutation are spared.

Trial at the Johns Hopkins Kimmel Cancer Center and other centers finds reduced-intensity haploidentical bone marrow ...