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Substrate reduction therapy is FDA approved and there is at least one treatment available on the market. [10] Gene therapy aims to replace a missing protein in the body through the use of vectors, usually viral vectors. [11] In gene therapy, a gene encoding for a certain protein is inserted into a vector. [11]
Severe weakness of the proximal muscles (shoulders, upper arms, thighs) on both sides of the body, very high blood levels of the enzyme creatine kinase (CK) being released by broken down skeletal muscle, and persistent symptoms and CK elevation despite stopping the offending statin medication are the hallmarks of SAAM.
Evans syndrome is an autoimmune disease in which an individual's immune system attacks their own red blood cells and platelets, the syndrome can include immune neutropenia. [1] [2] These immune cytopenias may occur simultaneously or sequentially.
Aspartate transaminase (AST) or aspartate aminotransferase, also known as AspAT/ASAT/AAT or (serum) glutamic oxaloacetic transaminase (GOT, SGOT), is a pyridoxal phosphate (PLP)-dependent transaminase enzyme (EC 2.6.1.1) that was first described by Arthur Karmen and colleagues in 1954.
AST is most commonly found in the liver, but also in significant amounts in heart and skeletal muscle. [ citation needed ] Measurement of ALT and AST were used in diagnosing heart attacks , although they have been replaced by newer enzyme and protein tests that are more specific for cardiac damage.
Platelet-rich plasma (PRP), also known as autologous conditioned plasma, is a concentrate of plasma protein derived from whole blood, centrifuged to remove red blood cells but retaining platelets. Though promoted for treating various medical conditions, evidence of its benefits was mixed as of 2020 , showing effectiveness in certain conditions ...
AST exists in two isoenzymes namely mitochondrial form and cytoplasmic form. It is found in highest concentration in the liver, followed by heart, muscle, kidney, brain, pancreas, and lungs. [10] This wide range of AST containing organs makes it a relatively less specific indicator of liver damage compared to ALT.
Ezetimibe/atorvastatin (trade names Liptruzet, Atozet) is a cholesterol lowering combination drug.In the United States, it was approved in May 2013, by the Food and Drug Administration for the treatment of elevated low-density lipoprotein (LDL) in patients with primary or mixed hyperlipidemia as adjunctive therapy to diet. [1]