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Lonafarnib

aberrant farnesylated form of lamin A called progerin in the inner nuclear membrane. … 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

Beremagene geperpavec

[L46916] DEB is caused by mutations in the _COL7A1_ gene that encodes collagen VII (COL7), a major component of the anchoring fibrils for dermal–epidermal cohesion. … Beremagene geperpavec is a live, replication-defective herpes simplex virus type 1 (HSV-1)-based vector therapy.

Ezogabine

It is a neuronal potassium channel opener being developed as a first-in-class antiepileptic drug (AED) and is currently being studied in Phase 3 trials as an adjunctive treatment for partial-onset seizures … Ezogabine (D23129) is a close structural analog of the centrally acting analgesic flupitrine.

Aurothioglucose

Contemporary research on the effect of gold salts treatment began in 1935, primarily to reduce inflammation and to slow disease progression in patients with rheumatoid arthritis [A32326].

Macitentan

Macitentan is a dual endothelin receptor antagonist used in the treatment of pulmonary arterial hypertension (PAH).[L35890] It was first approved by the FDA in 2013. … A combination product (Opsynvi) comprising macitentan and [tadalafil] was approved in Canada in October 2021 for the treatment of PAH.[L39105] It was subsequently approved by the FDA in March 2024.

Vigabatrin

Although administered as a racemic mixture, only the S(+) enantiomer is pharmacologically active. … [A202124] Its use is now generally reserved for patients who have failed alternative therapies, and its US approval by the FDA in 2009 mandated the creation of a drug registry to monitor patients for visual

Chloroform

It is a colorless, sweet-smelling, dense liquid that is produced on a large scale as a precursor to PTFE and refrigerants, but the latter application is declining. … Chloroform is an organic small molecule, member of the family of the chloromethanes that presents a formula of CHCl3.

Pyrophosphoric acid

Sacubitril

Sacubitril is a prodrug neprilysin inhibitor used in combination with valsartan to reduce the risk of cardiovascular events in patients with chronic heart failure (NYHA Class II-IV) and reduced ejection … It was approved by the FDA after being given the status of priority review for on July 7, 2015.

Echothiophate

A potent, long-acting irreversible cholinesterase inhibitor used as an ocular hypertensive in the treatment of glaucoma. Occasionally used for accomodative esotropia.