LSD Clinical Studies

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.

Sponsor: Protalix Biotherapeutics

A Multi Center Extension Study of PRX-102 Administered by Intravenous Infusions Every 2 Weeks for 24 Months to Adult Fabry Patients

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

Small molecule therapy development and clinical studies in lysosomal storage diseases

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.

Lysosomal Disease Bank

This is a specimen bank which aims to store cells, blood, urine and clinical data collected from patients diagnosed with lysosomal storage diseases.