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Sepiapterin

Sepiapterin is a small molecule activator of phenylalanine hydroxylase (PAH) used to reduce phenyalanine levels in patients with phenylketonuria. … It is a natural precursor of the enzymatic co-factor tetrahydrobiopterin (BH4), which activates PAH and helps reduce blood phenylalanine levels by enhancing the conformational stability of misfolded PAH

Mobocertinib

Mobocertinib is a kinase inhibitor targeted against human epidermal growth factor receptor (EGFR). … It is used specifically in the treatment of non-small cell lung cancer (NSCLC) caused by exon 20 insertion mutations in the _EGFR_ gene,[L38368] which are typically associated with a poorer prognosis (

Fosdenopterin

[A230088] Signs and symptoms begin shortly after birth and are caused by a build-up of toxic sulfites resulting from a lack of SOX activity. … 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.

Serdexmethylphenidate

[A230698, A230708] Serdexmethylphenidate is a prodrug of the CNS stimulant [dexmethylphenidate], a common first-line treatment for ADHD, that is combined with [dexmethylphenidate] to provide extended plasma … [L32298, L32323] Serdexmethylphenidate was granted FDA approval on March 2, 2021, and is currently marketed as a combination capsule with [dexmethylphenidate] under the trademark AZSTARYS™ by KemPharm

Durlobactam

Durlobactam is a diazabicyclooctane non-beta-lactam, beta-lactamase inhibitor. It is typically given in combination with [sulbactam] to protect it from degradation by certain serine-beta-lactamases.

Bromopyruvate

Crinecerfont

[A264818] Crinecerfont is a selective antagonist of corticotropin-releasing factor (CRF) type 1 receptor that works to reduce excessive ACTH secretion from the pituitary. … Patients with congenital adrenal hyperplasia (CAH) face two major problems: adrenal insufficiency, caused by insufficient endogenous cortisol production, and androgen excess, caused by a counter-regulatory

Benzgalantamine

Benzgalantamine is a prodrug of [galantamine]. … Gastrointestinal adverse effects are the most frequently reported side effects in patients undergoing treatment with cholinesterase inhibitors, including galantamine, and are often a reason for treatment

Chlorothymol

Chlorotymol is a [Thymol] derivative.

Parethoxycaine

Parethoxycaine is a local anesthetic that was previously approved in the US.