Sanfilippo syndrome patients present cardiac abnormalities with mild progression over time. However, these symptoms do not raise any specific…
Articles by Ashraf Malhas, PhD
Several approaches are currently being investigated to allow the delivery of novel therapeutic agents into the central nervous system and…
Cognitive and imaging data are sensitive markers that can be used to assess changes in the disease course of Sanfilippo…
Analyzing small molecules produced by cells in the blood of Sanfilippo syndrome patients can be used as a minimally invasive tool for…
A gene therapy led to the brain producing the proper form of an enzyme whose faulty version causes Sanfilippo syndrome type…