Several clinical studies related to the natural history of different LSD and early and late phase clinical trials in collaboration different pharmaceuticals. Most subjects are evaluated and recruited for the studies in clinic. These studies have Dr. Gustavo Maegawa MD, PhD as PI and with other research on going in University of Florida LSD Program.
This is an extension of previous phase I/II clinical trial to continue to assess the safety, tolerability and exploratory efficacy parameters of PRX-102 in adult patients with Fabry disease
The major goal is to study develop small molecules that can be used as therapeutic agents for lysosomal storage diseases. Secondly, the study aims to delineate the natural history of lysosomal storage disorders as well as determine potential biomarkers to evaluate these genetic disorders.
This is a specimen bank which aims to store cells, blood, urine and clinical data collected from patients diagnosed with lysosomal storage diseases.