Michela Luciano, PhD,  Science Writer—

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…

Tralesinidase alfa, also known as TA-ERT, Spruce Biosciences’ investigational enzyme replacement therapy (ERT) for children with Sanfilippo syndrome type B, has received breakthrough therapy designation from the U.S. Food and Drug Administration (FDA). The designation is intended to speed up the development and review of treatments that show early evidence…