medicine: cystagon

cystagon description








Cysteamine is the chemical compound with the formula HSCH2CH2NH2. It is the simplest stable aminothiol and a degradation product of the amino acid cysteine. It is often used as the hydrochloride salt, HSCH2CH2NH3Cl (CAS#[156-57-0])

Biochemical and pharmaceutical applications

Under the trade name Cystagon, cysteamine is used in the treatment of disorders of cystine excretion. cysteamine cleaves the disulfide bond with cystine to produce molecules that can escape the metabolic defect in cystinosis and cystinuria.

It is also used for treatment of radiation sickness.

Cysteamine is used in the body to form the essential biochemical Coenzyme A by combining with pantothenate and adenosine triphosphate.

In 2008, Raptor Pharmaceuticals started phase II clinical trials testing a delayed release (DR) preparation of cysteamine bitartrate for Huntington's Disease. DR cysteamine is also being investigated as a treatment for Cystinosis, Batten Disease, and NASH.

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