Familial hypercholesterolemia disorder in two generations simultaneously." "The use of total cholesterol alone may best discriminate between people with and without FH between the ages of 1 to 9 years." Screening of toddlers has been suggested, and results of a trial on 10,000 one-year-olds were published in 2016. Work was needed to find whether screening was cost-effective, and acceptable to families. FH is usually treated with statins. Statins act by inhibiting the enzyme hydroxymethylglutaryl CoA reductase (HMG-CoA-reductase) in the liver. In response, the liver produces more LDL receptors, which remove circulating LDL from the blood. Statins effectively lower cholesterol and LDL levels,
Specialty drugs in the United States drugs. Specialty drugs are more complex to manufacture. They are "highly complex medications, typically biology-based, that structurally mimic compounds found within the body". Specialty drugs are often biologics—"drugs derived from living cells"—but biologics are "not always deemed to be specialty drugs". Biologics "may be produced by biotechnology methods and other cutting-edge technologies. Gene-based and cellular biologics, for example, often are at the forefront of biomedical research, and may be used to treat a variety of medical conditions for which no other treatments are available." According to the U.S. Food and Drug Administration (FDA) biologics, or Some specialty drugs can be