Generalized lymphatic anomaly, or lymphangiomatosis, is a rare condition involving the abnormal overgrowth of lymphatic vessels in the lungs, pleura, bones, and soft tissue.
Myeloproliferative neoplasms (MPNs) are a rare, closely related group of blood disorders in which the bone marrow overproduces red blood cells, white blood cells, or platelets.
Pearson syndrome is a very rare condition that affects various parts of the body, and it is caused by a mutation — or changing of structure — in the mitochondrial...
Red blood cell (RBC) disorders are conditions that affect red blood cells, the cells of blood that carry oxygen from the lungs to all parts of the body.
Shwachman-Diamond syndrome (SDS) is a rare, inherited bone marrow failure, characterized by a low number of white blood cells, poor growth due to difficulty absorbing food, and, in some cases...
Sickle cell disease is an inherited blood disorder where cells cluster together, making it difficult for them to move through small blood vessels, thus denying oxygen to the blood.
White blood cell disorders can cause bone marrow to produce too many or too few white blood cells, or to produce white blood cells that do not function as they...
ZYNTEGLO™, also called beti-cel (betibeglogene autotemcel), is a gene therapy for children and adults with beta-thalassemia who require regular transfusions of red blood cells.