Novel Drug siRNA that lowers hypercholesterolemia in patients with gene disorder heterozygous familial hypercholesterolemia (HeFH) or clinical atherosclerotic cardiovascular disease (ASCVD)

Novel drug for hypercholesterolemia which is a SiRNA is now approved by USFDA for heterozygous familial hypercholesterolemia associated with genetic disorder

(inclisiran)  injection of Novartis a silensing RNA inhibits the process of translation of protein so that faulty enzyme production stops which is responsible for lipid production and hyperlipidemia

Basic constituent of the human body is a cell while proteins and fat are building material of a cell, synthesis of proteins and enzymes whichintern are responsible for cell wall and lipid (fat) synthesis) in our body this entire process is controlled through DNA transcription and translation process which involves mRNA, the drug (inclisiran) injection of Novartis.acts as a dummy mRNA and inhibit enzyme responsible for LDL-C cholesterol synthesis. 

A deficiency in the gene or unwanted defective gene expression causes synthesis of excessive or lower amounts of essential enzymes or proteins and lipids.

One example of such a disease is heterozygous familial hypercholesterolemia. 

Which is caused by defective gene expression which cant control synthesis of low density lipoprotein, because of which patients sufer from hypercholesterolemia and diseases associated with it clinical atherosclerotic cardiovascular disease (ASCVD)

Currently available approved drugs are statins which are not be tolarated for longer duration, patients have to take them for life long, and hence may not be as effective as required. 

Leqvio (inclisiran) injection Injection: 284 mg/1.5 mL is given at once under the skin after 3 moths one injection and after that once in six month injection, is to be given to patient, this therapy is required to be given along with diet control and maximum maximally tolerated statin therapy.

   

Drug (inclisiran) is indicated as an adjunct to diet and maximally tolerated statin therapy for the  of adults with heterozygous familial hypercholesterolemia (HeFH) or clinical atherosclerotic cardiovascular disease (ASCVD), who require additional lowering of low- density lipoprotein cholesterol (LDL-C) 

or alone or in combination with other lipid-lowering therapies in people who are statin-intolerant,

hypercholesterolemia (heterozygous familial and non-familial) or mixed dyslipidaemia

Process of inhibition of Translation through Si RNA that inhibits enzyme synthesis

Also see:

Orphan Drug approved for hypercholesterolemia due to genetic disorder

https://www.pharmacistspharmajournal.org/2013/01/new-drug-juxtapid-lomitapide-for.html

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