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Tay-Sachs, Canavan, GM1 and Sandhoff diseases

Pyrimethamine

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Pyrimethamine

Pyrimethamine is a drug is approved for treating malaria, and has also been found to have an effect on other diseases such as toxoplasmosis, a life-threatening parasitic infection. It has also been used as an off-label therapy for Tay-Sachs (Late Onset and Juvenile). Pyrimethamine is based on pharmacological chaperone technology.

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