General Info
Lysosomal Storage Disease
Sanfilippo Syndrome
Clinical Trials
Univ. Of Minn. Natural History Study
Intrathecal Enzyme Replacement Phase I/II Study
Intrathecal Enzyme Replacement Extension Study
Substrate Reduction/Optimization
Position Paper on the Use of Genistein
Zacharon Pharmaceuticals Paper on Their Substrate Optimization Therapy
Chaperon Therapy
Gene Therapy
General Research on the Disease
