Recent Posters and Abstracts
SSIEM – Society for the Study of Inborn Errors of Metabolism
Hamburg, Germany
September 2007
- PHARMACOLOGICAL CHAPERONE THERAPY FOR THE TREATMENT OF GAUCHER DISEASE: AT2101 INCREASES ACID β-GLUCOSIDASE LEVELS IN CELLS, MICE AND HEALTHY HUMAN SUBJECTS (poster)
- AT1001, A PHARMACOLOGICAL CHAPERONE DRUG CANDIDATE FOR THE TREATMENT OF FABRY DISEASE (poster)
ESHG European Human Genetics Conference
Nice, France
June 2007
- THE PHARMACOLOGICAL CHAPERONE AT1001 INCREASES LEVELS OF Α-GALACTOSIDASE A IN FABRY PATIENT CELL LINES AND REDUCES GL-3 LEVELS IN A FABRY TRANSGENIC MOUSE MODEL (Poster)
- THE PHARMACOLOGICAL CHAPERONE AT2101 INCREASES Β-GLUCOCEREBROSIDASE LEVELS IN MACROPHAGES AND LYMPHOBLASTS DERIVED FROM GAUCHER PATIENTS (Poster)
Society for Biomolecular Screening
Montreal, Canada
April 2007
American College of Medical Genetics
Nashville, Tennessee
March 2007
- THE PHARMACOLOGICAL CHAPERONE ISOFAGOMINE INCREASES L444P GCASE LEVELS IN MICE WITH A GAUCHER DISEASE-LIKE PHENOTYPE (Abstract)
- A NEW APPROACH TO THE TREATMENT OF GAUCHER DISEASE: MECHANISM OF ENHANCEMENT OF N370S GCASE ACTIVITY (Abstract)
- THE PHARMACOLOGICAL CHAPERONE, AT1001, INCREASES LEVELS OF MUTANT AND WILD TYPE Α-GALACTOSIDASE A, THE ENZYME DEFICIENT IN FABRY DISEASE, IN VITRO, IN VIVO, AND IN A PHASE 1 CLINICAL TRIAL (Abstract)
- PHASE 1 STUDIES OF THE SAFETY, PHARMACOKINETICS AND PHARMACODYNAMICS OF AT2101, A PHARMACOLOGICAL CHAPERONE FOR THE TREATMENT OF GAUCHER DISEASE (Abstract)
- PHASE 1 STUDIES OF THE SAFETY, PHARMACOKINETICS AND PHARMACODYNAMICS OF AT2101, A PHARMACOLOGICAL CHAPERONE FOR THE TREATMENT OF GAUCHER DISEASE (Poster)
- THE PHARMACOLOGICAL CHAPERONE AT1001 INCREASES LEVELS OF MUTANT AND WILD TYPE Α-GALACTOSIDASE A, THE ENZYME DEFICIENT IN FABRY DISEASE, IN VITRO, IN VIVO AND IN A PHASE 1 CLINICAL TRIAL
(Poster) - THE PHARMACOLOGICAL CHAPERONE ISOFAGOMINE INCREASES L444P GCASE LEVELS IN MICE WITH A GAUCHER DISEASE-LIKE PHENOTYPE (Poster)


