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JCI Reduced heparan sulfate levels in cerebrospinal fluid reflect brain neuron correction in Sanfilippo B mice

United States · United States

JCI Reduced heparan sulfate levels in cerebrospinal fluid reflect brain neuron correction in Sanfilippo B mice

Delivering a lab-made version of the missing enzyme in Sanfilippo syndrome type D directly into the brain reduced toxic heparan sulfate buildup, a key Sanfilippo event, and eased signs of inflammation in a mouse model of the disease, a new study reports. These benefits were observed even when the treatment was given on a schedule designed to mimic a real-world regimen in patients, “demonstrating preclinical efficacy for [Sanfilippo syndrome type…See the Story