Lysosomal and Rare Disorders Research and Treatment Center, Inc.

Fairfax, Virginia 22030

4 studies enrolling now · 11 studies all time

What they study most

Fabry Disease, Healthy, Late Onset Pompe Disease, Mucopolysaccharidosis II, Pompe Disease, Pompe Disease (Late-onset), Primary Immune Deficiency

Studies at this site

• A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease — Recruiting now
• A Study of Patients With Fabry Disease (US Specific) — Recruiting now
• A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants — Recruiting now
• A Global Prospective Observational Registry of Patients With Pompe Disease — Recruiting now
• A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease — Running, not enrolling
• A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease — Running, not enrolling
• Study to Assess the Safety, Tolerability, PK and PD of ABX1100 — Completed
• A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD) — Completed
• ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD — Running, not enrolling
• Study of Immune Globulin Intravenous (Human) GC5107 in Pediatric Subjects With Primary Humoral Immunodeficiency — Running, not enrolling
• Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II — Withdrawn before enrolling