Rapid Test Expected to Help in Developing Treatments for Sanfilippo

Apr 29, 2022 | News, Research

A new study shows a 30-minute, inexpensive, and easy-to-use test may help measure the activity of NDST1, one of the main enzymes involved in heparan sulfate, the sugar molecule that build ups and causes Sanfilippo syndrome.

Sanfilippo syndrome, or MPS III, is a genetic disorder wherein toxic levels of heparan sulfate (HS), a complex sugar molecule, accumulate and cause damage, especially to the brain.

Therapies that target HS production and avoid its accumulation are likely to ease Sanfilippo symptoms.

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