Lysosomal and Rare Disorders Research and Treatment Center, Inc

Summary

Lysosomal and Rare Disorders Research and Treatment Center, Inc is a medical facility located in Fairfax, Virginia. This center is recognized for care of Fabry Disease, Pompe Disease, Gaucher Disease, Primary Immunodeficiency, Gaucher's Disease and other specialties. Lysosomal and Rare Disorders Research and Treatment Center, Inc is involved with conducting 17 clinical trials across 15 conditions. There are 1 research doctors associated with this hospital, such as Ozlem Goker-Alpan, MD.

Top PIs

Clinical Trials running at Lysosomal and Rare Disorders Research and Treatment Center, Inc

Fabry Disease

Pompe Disease

Migalastat

for Fabry Disease

An open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric subjects 2 to \< 12 years of age with Fabry disease and with amenable GLA variants.

Recruiting

2 awards

Phase 3

3 criteria

PRX-102

for Fabry Disease

A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.

Recruiting

1 award

Phase 2 & 3

3 criteria

Migalastat

for Fabry Disease

This is an observational study to evaluate the effects of treatment on long-term effectiveness, safety, and health-related quality of life (HRQOL) in patients with Fabry disease, with a main focus on migalastat.

Recruiting

1 award

N/A

10 criteria