Clinical Trials & LSDs

Clinical Research

Clinical trials are an essential tool used by our researchers to unravel individuals' intricacies with LSDs and other rare genetic disorders.

LDRTC has been conducting clinical trials for over a decade. The crucial data collected by these studies ensures that our patients receive the best care.

We conduct industry-sponsored and investigator-initiated studies in the field of lysosomal disorders, including but not limited to Pompe, Fabry, Gaucher, MPS, and Niemann-Pick Diseases.

Our care facility also participates in the latest clinical trials, including gene therapy and other treatments for various other rare and chronic disorders that include MPS III, seasonal allergies, asthma, and primary immunodeficiency.

Dr. Ozlem Goker-Alpan is an expert in lysosomal and rare genetic disorders and oversees the clinical care of many individuals with these conditions at our Center. 
Who can participate?
Anyone diagnosed with a lysosomal or other rare disorder may be part of our studies. Each clinical trial has a medical objective, targeting either an individual with a specific condition or a particular group based on their age, sex, race, or ethnicity.

The safety of our volunteers is our number one priority. Our staff assists them throughout all phases of the trials. Once they apply for our trials, our team will conduct a screening with each volunteer and direct them to the most appropriate study.

Would you like to be part of our clinical trial?