Febuxostat and topiroxostat are second-generation xanthine oxidase inhibitors that reduce
uric acid production. Febuxostat came into broad use as a treatment for hyperuricemia and …

In α1 antitrypsin deficiency, a metabolic disorder caused by mutations in the SERPINA1
gene, mutant α1 antitrypsin polymerizes within hepatocytes, precluding it from being …

Research into treatments for viral infections has traditionally focused on viral targets. This is
also true for RNA viruses, where common targets include viral RNA-dependent RNA …

Obesity is associated with up-or downregulation of certain gene products relative to the
levels seen in individuals with a normal body weight, and blood levels for many of these …

The VEGF tyrosine kinase inhibitor, axitinib (Pfizer's Inlyta®, a follow-up to sunitinib, which
will lose patent exclusivity in 2021) is a component of the current gold standard treatment for …

Bromantane (adamantylbromphenylamine) is an anxiolytic psychostimulant developed and
used in Russia at doses of 50–100 mg for what used to be called neurasthenia. It also is a …

LINCL Type 2 (Jansky-Bielschowsky disease) is a lysosomal storage disorder from the
neuronal ceroid lipofuscinosis group that is often summarily referred to as Batten disease …

Desmopressin (1-desamino-8-D-arginine vasopressin; dDVAP) is a synthetic and
generically available antidiuretic peptide hormone that has been marketed for the treatment …

Nonsense-mediated decay protects the cell against the deleterious effects of aberrant
mRNA transcripts that contain premature termination codons or carry a RNA-destabilizing 6 …

Rotenone, a plant flavonoid insecticide and piscicide, exerts its toxic effect by inhibiting
mitochondrial electron transport from iron–sulfur centers in complex I to ubiquinone …