The National Organization for Rare Disorders is a coalition of patient advocacy groups and the first national nonprofit that represents patients and families affected by all rare diseases.

ALD protein is needed to clear toxic molecules called very long-chain fatty acids (VLCFAs) in the brain, and if mutated causes the VLCFAs to accumulate and damage the myelin sheath. Using eli-cel, ...

In addition, children with sickle cell anemia may also need a blood transfusion ... After femoral access is obtained, the arterial sheath is flushed with heparinized saline and connected to a flow ...

A patient on Long Island is the first in New York to be cured of sickle cell anemia, doctors say. They credit state-of-the-art genetic treatment. Medical history made at Cohen Children's Medical ...

Sickle cell disease is a genetic blood disorder characterised by the production of abnormal haemoglobin, which causes red blood cells to become rigid and sickle-shaped. These cells tend to clump ...

Attorneys for Bryan Kohberger plan to argue at trial that the knife sheath recovered from the crime scene could have been planted by the real killer of four University of Idaho students ...

However, they've got him with this, I think. Morgan believes DNA taken off *** bloody knife sheath is key to the case. They would have had to have searched this microscopically very, very ...