Faculty

Queenie Tan, M.D., Ph.D.

Clinical Studies

Clinical and Basic Investigations Into Congenital Disorders of Glycosylation Rochester, Minn.
The purpose of this study is to establish the prevalence and severity of specific morbid indicators of disease severity such as specific organ system involvement, degree of cognitive disability, and case-fatality associated with various congenital disorders of glycosylation (CDG), and establish a dynamic platform to effectively disperse clinically relevant findings to families, non-expert clinicians and researchers, as well as provide a verified method to link these individuals to experts in CDG.

Clinical and Basic Investigations Into Congenital Disorders of Glycosylation Jacksonville, Fla.
The purpose of this study is to establish the prevalence and severity of specific morbid indicators of disease severity such as specific organ system involvement, degree of cognitive disability, and case-fatality associated with various congenital disorders of glycosylation (CDG), and establish a dynamic platform to effectively disperse clinically relevant findings to families, non-expert clinicians and researchers, as well as provide a verified method to link these individuals to experts in CDG.

A Phase 2, Randomized, Open-Label, 24-Week Study to Assess the Pharmacodynamics, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of GLM101 Administered Intravenously to Adult Participants with PMM2-CDG Rochester, Minn.
The purpose of this study is to evaluate changes in coagulation and antithrombosis factors in adult participants with PMM2-CDG after 24 weeks of dosing.
A Prospective, Randomized, Double-Blind, Placebo-Controlled, Two-Center study of Epalrestat Therapy in Pediatric Subjects with Phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG) Rochester, Minn.
The purpose of this study is to evaluate the clinical and metabolic improvement of pediatric subjects with PMM2-CDG who are treated with epalrestat therapy compared to placebo, and to evaluate the safety, clinical improvement, and pharmacokinetics (PK) of epalrestat therapy in pediatric patients with PMM2-CDG compared to placebo when compared to baseline.