phosphocysteamine การใช้

A study in the April issue of the journal Nature Medicine indicates that the drug phosphocysteamine may be useful in treating INCL victims.
Phosphocysteamine has been used for 20 years in the treatment of cystinosis, a rare hereditary disorder that, if untreated, results in blindness, kidney failure and death by about age 9.
Various salt forms have been worked with in labs, in clinical trials, and in marketed drugs, including cysteamine hydrochloride, phosphocysteamine, and cysteamine bitartrate; marketed capsules are the cysteamine bitartrate salt.
A team led by Anil B . Mukherjee of NICHD tested laboratory cultures of cells taken from patients with INCL . They found that phosphocysteamine reduced the amount of protein-fat complexes within the cells to minimal levels.