New drug for atypical Hemolytic Uremic Syndrome (aHUS), which blocks the protein responsible for breakdown of RBC which lead to sever lifethreatening conditions like stroke and renal failure.

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)

Also see

what is heamolytic anemia ?

Why Betalactam Antibiotics require separate manufacturing aria in a pharmaceutical manufacturing company?

Transdermal drug delivery system new requirements for quality and for regulatory submissions

Good Manufacturing Practices

Liposome drug delivery system

A new vaccine for TB which can provide protection in late stage infection.

What is clinical trial protocol?

What is an artificial pacemaker device

Therapeutic Index (Therapeutic Ratio) of a drug? What is Meant by Narrow Therapeutic Index?

Microbial load limits for water for pharmaceutical use

Ultraviolet light as antimicrobial disinfectant in water for pharmaceutical use

Why water for pharmaceutical use is always kept in close loop in continuous circulation?

Ion exchange resin and its application in pharmaceutical dosage forms, and drug delivery systems.

Why Betalactam Antibiotics require a separate manufacturing aria in a pharmaceutical manufacturing company?

Chemistry behind the allergic reactions and mode of action of betalactum antibiotics

US FDA approved first oral drug to reduce Multiple Sclerosis MS relapses

US FDA limits dosage of acetaminophen to 325 mg per unit dosage form,Requires boxed warning on lables.

US FDA honours woman who rejected thalidomide and saved millions of children’s from thalidomide

A Pharmaceutical exceipent Beta Cyclodextrin being used in a life saving treatment

Nanoparticles of insulin in place of painful injections in treatment for diabetes patients, transdermal , nasal drug delivery systems

Corifact becomes the first drug to treat congenital deficiency of Factor XII.

Protein which is responsible for cancer spread discovered An antisense RNA treatment or a drug could be designed to treat cancer efficiently and safely.

Novel Drugs: Cancer Chemotherapy Using Nanoparticles May Reduce Harmful Side Effects.

What is Nanotechnology, its applications in medicine and pharmaceuticals, drug developments?

%d bloggers like this: