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Pharmacological chaperones can also help patients living with other CDG, as long as the defective protein still has some (even if small) ability to work.
How can Pharmacological chaperones help people living with PMM2-CDG?
A library of chemical compounds was studied to check if any of them could help the PMM2 protein function better. One compound named 1-(3-chlorophenyl)-3-3-bis(pyridine-2-yl)urea) was tested in a cellular model with good results. However, in high amounts, this compound was toxic to the cells. Now, the researchers are working to change the structure of the compound to make it better (non-toxic).