Dialog Box


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.


The University of Manchester is currently conducting a phase III double-blinded, placebo-controlled clinical trial of high-dose oral Genistein.


Genistein is a natural substance found in some foods such as soybeans. 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 if Genistein can slow the progression of the disease in children with Sanfilippo.

The trial is funded by the MPS Society UK.


For more information, visit MPS Society UK