Pioneering Clinical Research for Rare Diseases

Clinical trials are the cornerstone of medical advancement, playing a pivotal role in developing treatments for various medical conditions. This is particularly true for Rare Diseases, including Lysosomal  Disorders (LDs). While significant progress has been made, many rare diseases still lack effective treatments. Moreover, existing therapies for some conditions may not address all aspects of the disease. Our goal through clinical research at LDRTC is twofold: to contribute to the development of novel treatments for diseases currently without therapy and to enhance existing treatment options where gaps in care exist. 


For over a decade, LDRTC has been at the forefront of clinical research in the Lysosomal disease area.

Our focus encompasses a wide range of Lysosomal Storage Disorders, including but not limited to Pompe, Fabry, Gaucher, Mucopolysaccharidoses (MPS), and Niemann-Pick Disease. We engage in both industry-sponsored and investigator-initiated studies, bringing together expertise and innovation to tackle these challenging conditions.

Our Clinical Trials Research Department is actively involved in conducting the latest clinical trials, including groundbreaking Gene Therapy studies. We take pride in our direct involvement in research that has led to FDA approval of numerous drugs. These drugs now serve as effective therapies, not only for our patients but also for individuals affected by these rare diseases globally.

At LDRTC, we understand the critical need for advanced treatments in the rare disease community. Our commitment to clinical trials research reflects our dedication to improving patient outcomes and quality of life. We strive to be at the cutting edge of medical research, offering new hope and possibilities through our pioneering clinical trials for patients both nationally and internationally.

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?