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Substrate Reduction Therapy

 

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

 

The objective of SRT for Sanfilippo is to help reduce the amount complex sugars or GAGs (which is the substrate) produced by the body so there is less to accumulate in the cells.

Genistein is a natural substance found in some foods such as soybeans. It is non-toxic and is relatively cheap, and is able to reduce the amount of complex sugars stored in the brains of mice with Sanfilippo Syndrome and improve brain function.

 

The University of Manchester conducted a phase III double-blinded, placebo-controlled clinical trial of high-dose oral genistein aglycone that concluded in 2018.

 

Unfortunately, no clinically meaningful benefit was seen for the 21 children in the trial. No further clinical trials with this product are planned.

 

For more information, please see statement from the University of Manchester