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Pariglasgene brecaparvovec

continued approval potentially contingent on confirmatory trials. … [L64050] Restoring hepatic G6Pase activity supports the release of glucose during fasting, and in clinical studies reduced patients' reliance on dietary cornstarch used to prevent hypoglycemia.

Corticotropin

It is an important player in the hypothalamic-pituitary-adrenal axis.

Fosdenopterin

In 2009 a recombinant, E. coli-produced cPMP was granted orphan drug designation by the FDA, becoming the first therapeutic option for patients with MoCD type A. … Molybdenum cofactor deficiency (MoCD) is an exceptionally rare autosomal recessive disorder resulting in a deficiency of three molybdenum-dependent enzymes: sulfite oxidase (SOX), xanthine dehydrogenase

Bismuth subsalicylate

Bismuth subsalicylate is an antacid and anti-diarrheal agent. … [A230728] It is an active ingredient found in Pepto-Bismol, a common over-the-counter medication that is used to temporarily treat discomforts of the stomach and gastrointestinal tract.

Palovarotene

Fibrodysplasia Ossificans Progressiva (FOP), with an estimated worldwide prevalence of one in 2 million individuals, is an exceptionally rare genetic disorder. … [A244920] Palovarotene was approved for use in Canada in January 2022 for the management of heterotopic ossification (HO) in patients with FOP, representing the first global approval for any FOP therapy

Rezafungin

[L45633,L45658] It was subsequently approved in the EU in November of the same year. [L52745] … Rezafungin is an echinocandin antifungal drug.

Ferrous cysteine glycinate

Metreleptin

in food intake. … [L41315] In February 2014, metreleptin was approved by the FDA for the treatment of complications of leptin deficiency, as an adjunct to diet, in patients with congenital generalized or acquired generalized

Saquinavir

In 1995 it became the first protease inhibitor approved by the FDA, followed shortly by ritonavir in 1996, and remains in clinical use today due to a relatively benign adverse effect profile as compared … Saquinavir is an HIV-1 protease inhibitor used in combination with [ritonavir] and other antiretrovirals for the treatment of human immunodeficiency virus-1 (HIV-1) infection.

Meningococcal polysaccharide vaccine group C

*N. meningitidis* is a bacteria that causes endemic and epidemic diseases including meningitis and meningococcemia. … It is subcutaneously administered as an active immunization against the invasive meningococcal disease caused by the serogroup C.