N-acetylcysteine Pharmacology and Applications in Rare Diseases-Repurposing an Old Antioxidant.

ABSTRACT: N-acetylcysteine (NAC), a precursor of cysteine and, thereby, glutathione (GSH), acts as an antioxidant through a variety of mechanisms, including oxidant scavenging, GSH replenishment, antioxidant signaling, etc. Owing to the variety of proposed targets, NAC has a long history of use as a prescription product and in wide-ranging applications that are off-label as an over-the-counter (OTC) product. Despite its discovery in the early 1960s and its development for various indications, systematic clinical pharmacology explorations of NAC pharmacokinetics (PK), pharmacodynamic targets, drug interactions, and dose-ranging are sorely limited. Although there are anecdotal instances of NAC benefits in a variety of diseases, a comprehensive review of the use of NAC in rare diseases does not exist.

SUBMITTER: Sahasrabudhe SA 

PROVIDER: S-EPMC10376274 | biostudies-literature | 2023 Jun

REPOSITORIES: biostudies-literature