GI-tract pain is likely caused by accumulation of lipids in the small vasculature of the GI tract, which obstructs blood flow and causes pain. Kidney
Kidney complications are common and serious effects of the disease; chronic kidney disease and kidney failure may worsen throughout life. The presence of protein in the urine (which causes foamy urine) is often the first sign of kidney involvement. End-stage kidney failure in those with Fabry disease typically occurs in the third decade of life, and is a common cause of death due to the disease. Heart
Fabry disease can affect the heart in several ways. The accumulation of sphingolipids within heart muscle cells causes abnormal thickening of the heart muscle or hypertrophy. This hypertrophy can cause the heart muscle to become abnormally stiff and unable to relax, leading to a restrictive cardiomyopathy causing shortness of breath.Fabry disease can also affect the way in which the heart conducts electrical impulses, leading to both abnormally slow heart rhythms such as complete heart block, and abnormally rapid heart rhythms such as ventricular tachycardia. These abnormal heart rhythms can cause blackouts, palpitations, or even sudden cardiac death.Sphingolipids can also build up within the heart valves, thickening the valves and affecting the way they open and close. If severe, this can cause the valves to leak (regurgitation) or to restrict the forward flow of blood (stenosis). The aortic and mitral valves are more commonly affected than the valves on the right side of the heart.
Abametapir, sold under the brand name Xeglyze, is a medication used for the treatment of head lice infestation in people six months of age and older.The most common side effects include skin redness, rash, skin burning sensation, skin inflammation, vomiting, eye irritation, skin itching, and hair color changes.Abametapir is a metalloproteinase inhibitor. Abametapir was approved for medical use in the United States in July 2020. The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication. Medical uses
Abametapir is indicated for the topical treatment of head lice infestation in people six months of age and older. Contraindications
Abametapir has no contraindications according to the labeling. Adverse effects
Common adverse effects are burning skin sensations (in 3% of patients), contact dermatitis (2%), skin redness (4%), rash (3%), and vomiting (2%). Interactions
Abametapir blocks the liver enzymes CYP3A4, CYP2B6 and CYP1A2 in vitro. A single application of the drug may lead to increased blood concentrations of drugs that are metabolized by these enzymes. Pharmacology
Mechanism of action
The drug inhibits enzymes called metalloproteinases. In lice, these enzymes play a role in egg development and survival; and consequently, blocking them will disrupt the lices life cycle. Pharmacokinetics
After application to the scalp, part of the substance reaches the bloodstream, where most of it (91.3–92.3%) is bound to plasma proteins. It is metabolized primarily by the liver enzyme CYP1A2 to abametapir hydroxyl and further to abametapir carboxyl (see structure drawings).