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Cysteamine

Cystinosis is a rare disease caused by mutations in the CTNS gene that encodes for cystinosin, a protein responsible for transporting cystine out of the cell lysosome. … [L15606] Cysteamine eye drops are a practical and effective option for those affected by ocular cystinosis.

NADH

It forms NADP with the addition of a phosphate group to the 2' position of the adenosyl nucleotide through an ester linkage. (Dorland, 27th ed) … It is found widely in nature and is involved in numerous enzymatic reactions in which it serves as an electron carrier by being alternately oxidized (NAD+) and reduced (NADH).

Methscopolamine

The oral tablets are used as an adjunct therapy for the treatment of peptic ulcer and is shown to be effective in decreasing the rate of recurrence of peptic ulcers as well as preventing complications. … Methscopolamine is a quaternary ammonium derivative of scopolamine and antagonist at muscarininc (mACh) receptors.

Vitis vinifera seed

Asfotase alfa

Hypophosphatasia is almost always fatal when severe skeletal disease is obvious at birth. … Asfotase alfa is a first-in-class bone-targeted enzyme replacement therapy designed to address the underlying cause of hypophosphatasia (HPP)—deficient alkaline phosphatase (ALP).

Serdexmethylphenidate

[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 … [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

Pitavastatin

as well as the need for surgical revascularization or angioplasty following a heart attack. … Pitavastatin, also known as the brand name product Livalo, is a lipid-lowering drug belonging to the statin class of medications.

Apadamtase alfa

[A262076] Patients with cTTP have a severe deficiency of a plasma metalloproteinase called ADAMTS13 (a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13), which is responsible … [A262071] Apadamtase alfa, sold under the brand name Adzynma (ADAMTS13, recombinant-krhn), was approved by the FDA in November 2023 for use as an enzyme replacement therapy in patients with cTTP.

Acetylcholine

A neurotransmitter. … Acetylcholine in vertebrates is the major transmitter at neuromuscular junctions, autonomic ganglia, parasympathetic effector junctions, a subset of sympathetic effector junctions, and at many sites in

Povidone

Povidone, also known as polyvinylpyrrolidone (PVP) or polyvidone, is a synthetic water-soluble polymer made from the monomer N-vinylpyrrolidone used as a binder in many pharmaceutical tablets and lubricant … It is also used in many technical applications with various roles as an adhesive, additive, and emulsifier.