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Patisiran

Patisiran is a first in class short interfering RNA for the treatment of patients with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis [L4220]. … The approval for Onpattro was granted to Alnylam Pharmaceuticals, Inc. in August of 2018. Onpattro has been granted Fast Track, Priority Review and Breakthrough Therapy, and Orphan Drug designations.

Aminolevulinic acid

A compound produced from succinyl-CoA and glycine as an intermediate in heme synthesis. It is used as a photochemotherapy for actinic keratosis. [PubChem]

Cholestyramine

Cholestyramine resin is quite hydrophilic, but insoluble in water. … Cholestyramine or colestyramine is a bile acid sequestrant. Bile acid sequestrants are polymeric compounds which serve as ion exchange resins.

Pyrophosphoric acid

Gallium Ga-68 gozetotide

positive lesions in men with prostate cancer. … [L43542] In December of the same year, the drug was fully authorized by the EMA.[L44682] In Octer 2022, Ga-68 PSMA-11 was approved by Health Canada for diagnostic use in men with prostate cancer.

Ataluren

Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and older who are able to walk. … More specifically, ataluren is used in the small group of patients whose disease is caused by a specific genetic defect (called a ‘nonsense mutation’) in the dystrophin gene.

Inulin

A starch found in the tubers and roots of many plants. Since it is hydrolyzable to fructose, it is classified as a fructosan. … It has been used in physiologic investigation for determination of the rate of glomerular function.

Nitrous oxide

At room temperature, it is a colorless non-flammable gas, with a pleasant, slightly sweet odor and taste. It is used in surgery and dentistry for its anesthetic and analgesic effects. … It is known as "laughing gas" due to the euphoric effects of inhaling it, a property that has led to its recreational use as an inhalant drug.

Thimerosal

Although thimerosal is banned in several countries, it continues to be included as a preservative in some vaccines in the United States and many vaccines in the developing world [L1685]. … Developed in 1927, thimerosal has been and is still being used as a preservative in some cosmetics, topical pharmaceuticals, and biological drug products, which includes vaccines.

Diethylamino hydroxybenzoyl hexyl benzoate

Diethylamino hydroxybenzoyl hexyl benzoate was approved in Europe in 2005, and is also marketed in the U.S., South America, Mexico, Japan and Taiwan. … It is used in concentrations up to 10% in sunscreen products, either alone or in combination with other UV absorbers [F107].