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Substrate Reduction Therapy


University of Manchester's MPSIII Genistein Trial (UK) - Sanfilippo Types A, B, C and D

The University of Manchester is currently conducting a phase III double-blinded, placebo-controlled clinical trial of high-dose oral Genistein Aglycone.  Previous research done by Pr Wegrzyn in Poland had concluded that Genistein had a potential efficacy for Sanfilippo patients at high dosage. A compound of Genistein is found in soy foods; it is non-toxic and is relatively cheap. It has already been found to reduce the amount of complex sugars stored in the brains of mice with Sanfilippo Syndrome and improve brain function. This human trial will determine the correct and safe dose, the effect of the drug on delaying the progression of the disease and how it improves symptoms.


The trial is funded by the MPS Society UK.


For more information, visit MPS Society UK