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Lonafarnib

[A224379, A224384, A224389, A224394, A224399] Mechanistically, HGPS is underpinned by a single heterozygous C-to-T mutation at position 1824 of the _LMNA_ gene, which results in the accumulation of an … Hutchinson-Gilford progeria syndrome (HGPS) is a rare autosomal dominant disorder estimated to affect approximately one in 20 million individuals resulting in adverse symptoms associated with premature

Cantharidin

Cantharidin is a naturally occurring odorless, colorless fatty substance of the terpenoid class that is produced as an oral fluid in the alimentary canal of the male blister beetle. … inflammatory model or in cancer treatment.

Pyrantel

Pyrantel was initially described in 1965 by researchers from Pfizer who sought cyclic amidines with suitable pharmacokinetic properties (specifically, duration of action) for use as an anthelmintic drug … A depolarizing neuromuscular-blocking agent causing longstanding nicotinic receptor activation, resulting in spastic paralysis of susceptible nematodes (worms).

Sodium tartrate

Sodium tartrate is generally recognized as safe (GRAS) as a direct human food ingredient. It acts as an emulsifier and pH control agent in food products [L2590]. … This compound is commonly used as an emulsifier in cheese/cheese spread products and is not to exceed 4% concentration, according to Health Canada regulations [L2594].

Trolamine

Trolamine contains small amounts of diethanolamine and ethanolamine and may also act as an antioxidant against the auto-oxidation of animal and vegetable fats [A27174]. … Trolamine, which is also referred to as triethanolamine (TEA), is a tertiary amine and a triol. It is a bifunctional compound that exhibits both properties of alcohols and amines.

Hexylresorcinol

It can be found in topical applications for minor skin infections and in oral solutions or throat lozenges for pain relief and first aid antiseptic. … The compound may also be used commonly in various commercial cosmetic anti-aging creams while ongoing studies research the possibility of using hexylresorcinol as an anti-cancer therapy - indications all

Retlirafusp alfa

Sebetralstat

This plasma kallikrein inhibitor works by reducing bradykinin, the substance that causes the characteristic swelling in HAE attacks [L53533]. The U.S. … Hereditary angioedema (HAE) is a rare genetic disease that causes sudden, painful swelling episodes in various body locations that can be life-threatening, particularly when affecting the throat [L53533

Nusinersen

An antisense oligonucleotide that induces survival motor neuron (SMN) protein expression, it was approved by the U.S. … FDA in December, 2016 as Spinraza for the treatment of children and adults with spinal muscular atrophy (SMA). It is adminstrated as direct intrathecal injection.

Temafloxacin

Temafloxacin is an antibiotic agent belonging to the fluoroquinolone drug class. … It was first approved for use in the U.S. market in 1992, but was withdrawn shortly due to the reports of serious adverse reactions, such as allergic reactions and hemolyric anemia, resulting in three