Atypical Hemolytic Uremic Syndrome (aHUS ) disease is causes because of genetic disorder because of which uncontrolled breakdown of red blood cells occur (RBCs) leading to anemia ,which does not only stops at that but further develops in to severe life threatening conditions like kidney failure and thromboembolic conditions, which may cause stroke and paralysis too.
Atypical Hemolytic Uremic Syndrome (aHUS ) affects mostly children and it is a rare disease.
There are very few treatments available for (aHUS ), now US FDA has approved a drug Soliris (eculizumab) which can now used to treat atypical Hemolytic Uremic Syndrome(aHUS).
How atypical hemolytic uremic syndrome is caused:
C5 protein is a protein which is a part of complement system of our immune system, it fix to antibodies and help in phagocytosis or destruction of unwanted infected cells or an unwanted antigen in our body .
In case of individual affected with atypical Hemolytic Uremic Syndrome C5 protein formed is abnormal due to mutation in gene which produce C5 protein and thus malformed C5 (complement system) causes destruction of RBCS in person affected with atypical Hemolytic Uremic Syndrome (aHUS ) .
How drug Soliris (eculizumab) act:
Drug Soliris (eculizumab) consist of humanized monoclonal antibodies IgG4 , which work against malformed complement system protein the C5 protein, it binds with it and stop its destructive action on RBCs.
Earlier solaris was also approved for treatment of paroxysmal nocturnal hemoglobinuria (PNH), where a affected persons red blood cells are destroyed and red urine is formed due to excretion of hemoglobin in urine. Drug Soliris (eculizumab) is classified as an orphan drug
The results out of clinical trial of drug Soliris (eculizumab) was able to demonstrate that it improves condition of kidney and also other blood count were improved and plate late count too was increased.
Side effects of drug Soliris (eculizumab):
Are as follows : High BP , infections in upper respiratory tract, infection of urinary tract diarrhea, vomiting , nausea and headache and decreased wbc count.
Alexion Pharmaceuticals in Cheshire, Conn market this Drug Soliris (eculizumab)
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