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Migalastat

This enzyme is responsible for breaking down glycosphingolipid substrate that, when deficient in patients with Fabry disease, builds up in the blood vessels, the kidneys, the nerves, the heart, and other … [L47036,L47057,L4274,L4278] In the U.S., it is estimated that more than 3,000 people are living with Fabry disease, and an estimated more than 50 percent of these diagnosed patients are currently untreated

Efgartigimod alfa

Efgartigimod alfa for intravenous use was granted FDA approval on December 17, 2021[L39501] and European Commission approval on August 11, 2022 for use in patients with myasthenia gravis. … and a reduction in electrical nerve impulses.

Influenza B virus B/Phuket/3073/2013 recombinant hemagglutinin antigen

Inactivated virus cannot replicate, and therefore cannot cause disease from infection, even in immunocompromised individuals. … In contrast, live vaccines are produced from "wild-type" or disease-causing viruses that have been attenuated, or weakened, through various laboratory techniques.

Triamterene

[L6163] Triamterene was approved by the Food and Drug Administration in the U.S. in 1964. … It works by promoting the excretion of sodium ions and water while decreasing the potassium excretion in the distal part of the nephron in the kidneys by working on the lumenal side.

Isosulfan blue

Thiosulfuric acid

Thiosulfuric acid is available in its salt forms sodium thiosulfate and sodium thiosulfate pentahydrate. … [A252827] Sodium thiosulfate is rapidly degraded in the stomach; therefore, it is normally administered through other routes, such as intravenous or topical.

Hydrogen peroxide

Hydrogen peroxide is an unstable compound in the presence of a base or catalyst, and is typically stored with a stabilizer in a weakly acidic solution. … Hydrogen peroxide is also considered as a generally recognized as safe compound by the FDA [L2024]; it is used as an antimicrobial agent in starch and cheese products, and as an oxidizing and reducing

Trifarotene

Trifarotene is a topical retinoid cream used in the treatment of acne vulgaris that was first approved for use in the United States in October 2019. … medications structurally and functionally analogous to [vitamin A], though later generation retinoids such as trifarotene and [adapalene] bear little structural resemblance to vitamin A and are analogous only in

Protein C

Once in its activated form, APC functions as a serine protease with potent anticoagulant effects, especially in the presence of its cofactor protein S. … A lack of protein C in the body would lead to unchecked coagulation activation, resulting in thrombin generation and intravascular clot formation.

Flutemetamol (18F)

It is used to evaluate Alzheimer's disease (AD) or other causes of cognitive decline as an Aβ-specific positron emission tomography (PET) ligand.[L35029] … Flutemetamol (18F), an analogue of [(11)C]PIB,[A18519] is a radioactive diagnostic drug used for Positron Emission Tomography (PET) imaging of the brain to estimate β amyloid neuritic plaque density.