The U.S. Food and Drug Administration approved
a new drug on Friday that reduces the complications associated with
sickle cell disease — the first drug approved for the blood disorder in
more than 20 years.
The drug, called Endari, consists of
L-glutamine, which is an amino acid, and is approved for sickle cell
patients five years and older.
"I am hoping we are finally seeing channels
opening, and that this will be the first of many new drugs to hit the
market [for sickle cell disease]," said Dr. Alexis A. Thompson, head of
the Hematology Section and Director of the Comprehensive Thalassemia
Program at the Ann and Robert H. Lurie Children’s Hospital of Chicago.
Sickle cell disease,
which predominately affects African-Americans, Latinos and other
minority groups, is an inherited lifelong disorder in which red blood
cells that are normally round and disc-shaped are instead crescent or
sickle-shaped due to abnormal hemoglobin.
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