Lorne Clarke - Heparin cofactor II-thrombin complex as an MPS disease biomarker:  Are the MPSs serpinopathies?
The Child and Family Research Institute, Vancouver, CANADA

Douglas Martin - Gene therapy of feline gangliosidosis with AAV vectors.
Auburn University , Auburn, Alabama

Takashi Shimada - Local and systemic gene therapy for metachromatic leukodystrophy.
Nippon Medical School, Tokyo, JAPAN

John Keimel - Intrathecal continuous or intermittent bolus delivery of laronidase results in elevated levels in canine CNS tissue.
Medtronic, Inc., Shoreview, Minnesota

C. Ronald Scott - Screening of newborns for lysosomal storage diseases.
University of Washington, Seattle, Washington

Joseph Orsini - Newborn screening for Krabbe disease.
New York State Dept. of Health, Wadsworth Center, Albany, New York

Olaf Bodamer - Newborn screening for lysosomal storage diseases – a reality?
University Children's Hospital, Vienna, AUSTRIA

Joan Keutzer - LSD enzyme assays in dried blood spots – from research to newborn screening.
Genzyme Corporation, Framingham, Massachusetts

William Wilcox - Females with X-linked Fabry disease frequently have significant organ involvement.
Cedars-Sinai Medical Center, Los Angeles, California

Elsa Shapiro - A proposed multicenter study of CNS structure and function in MPS I.
University of Minnesota, Minneapolis, Minnesota

Christine Eng - Biochemical and molecular genetics diagnostic laboratories network.
Baylor College of Medicine, Houston, Texas

Lynda Polgreen - Endocrine abnormalities in patients with mucopolysaccharidoses.
University of Minnesota, Minneapolis, Minnesota

Mia Horowitz - ER retention and degradation as the molecular basis underlying Gaucher disease heterogeneity.
Tel Aviv University, Tel Aviv, ISRAEL

Elena L. Aronovich - Sleeping Beauty Transposon-mediated correction of Mucopolysaccharidosis Type I.
University of Minnesota, Minneapolis, Minnesota

Christiane Auray-Blais - Development of a filter paper method applicable to a mass urinary screening for Fabry disease.
Université de Sherbrooke, Sherbrooke, Quebec, CANADA

Michael Beck - The Hunter Outcome Survey (HOS): A registry of Mucopolysaccharidosis II patients.
Childrens Hospital University of Mainz, Mainz, Rhineland-Palatinat, GERMANY

Kendra J. Bjoraker - Quality-of-life and psychosocial outcomes in patients with MPS disorders who have undergone hematopoietic stem cell transplant.
University of Minnesota, Minneapolis, Minnesota

Roscoe O. Brady - Current and future therapies for hereditary metabolic disorders.
National Institute of Neurological Disorders and Stroke, Bethesda, Maryland

Sara Cathey - Mucolipidoses II and III: Clinical and molecular characterization.
Greenwood Genetic Center, Greenwood, South Carolina

Francis Choy - Type II perinatal lethal Gaucher disease: Identification of a novel mutation, P332L, in a case study from Vancouver Island, British Columbia, Canada.
University of Victoria, Victoria, British Columbia, CANADA

Joseph Clarke - Alternative dosing regimens of agalsidase alfa (Replagal®) in Fabry disease.
Hospital For Sick Children, Toronto, Ontario, CANADA

Paul Harmatz - Cardiac findings in MPS VI patients after enzyme replacement therapy.
Children's Hospital & Research Center Oakland, Oakland, California

C.E.M. Hollak - A prospective study of neurological manifestations and other comorbidities in adult Type 1 Gaucher disease.
Academic Medical Centre, Amsterdam, North Holland, THE NETHERLANDS

Robert J. Hopkin - Quality of life for pediatric patients with Fabry disease.
Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio

John Jennings Jr. - Mitochondrial abberrrations and increased cell death in Mucolipidosis IV.
University of Pittsburgh, Pittsburgh, Pennsylvania

Arthur Johnson - Molecular basis for unusually low alpha-L-iduronidase activity in normal individuals.
University of Minnesota, Minneapolis, Minnesota

Mohammad Hassan Karimi-Nejad - Sixteen years of experience of biochemical analysis and prenatal diagnosis of lysosomal storage disease in Iran.
Karimi-Nejad-Najmabadi Pathology and Genetic Center, Tehran, IRAN

John G. Keimel - Intrathecal continuous or intermittent bolus delivery of laronidase results in elevated levels in canine CNS tissue.
Medtronic, Inc, Shoreview, Minnesota

Dwight D. Koeberl - Enhanced response to enzyme replacement therapy in Pompe disease following the induction of immune tolerance.
Duke University Medical Center, Durham , North Carolina

Jennifer M. Kwon - Quantifying progression of Batten disease: Development and use of a reliable and valid scale.
University of Rochester Medical Center, Rochester, New York

Abby KC Lau - Molecular investigations of a novel mutation in the IDS gene and an iduronate-2-sulfatase mutant without the first 33 amino acids.
The Chinese University of Hong Kong, Hong Kong, CHINA

Don Mahuran - Pyrimethamine as a potential pharmacological chaperone for late-onset forms of GM2 Gangliosidosis.
Hospital For Sick Children, Toronto, Ontario, CANADA

Kerry McEachern - Genz-112638: A novel orally available ceramide-based inhibitor of glucosylceramide synthase for treating Gaucher disease.
Genzyme Corporation, Framingham, Massachusetts

Colleen McNeil - Hematopoietic stem cell transplantation (HSCT) and enzyme replacement therapy (ERT) in a patient with Mucopolysaccharidosis Type I, Hurler syndrome.
Alberta Children's Hospital, Calgary, Alberta, CANADA

Makoto Migita - An asymptomatic heterozygous female with Fabry disease -implications for enzyme replacement therapy.
Nippon Medical School, Bunkyo-ku, Tokyo, JAPAN

Greg Pastores - Beneficial effects of miglustat on skeletal symptoms in Type I Gaucher disease: a meta-analysis.
New York University School of Medicine, New York, New York

Marc Patterson - Miglustat in Niemann-Pick type C (NPC) disease: results of 12-months treatment.
Columbia University, New York, New York

Lynda Polgreen - Growth and the use of growth hormone in patients with Hurler syndrome following hematopoietic stem cell transplantation.
University of Minnesota, Minneapolis, Minnesota

Katherine Ponder - Aortic disease in Mucopolysaccharidosis I mice is due to increased expression of matrix metalloproteinase 12 and cathepsin S.
Washington University School of Medicine, St. Louis, Missouri

Sherri L. Sandberg - Treatment of multiple sulfatase deficiency with recombinant human arylsulfatase B.
University of Minnesota, Minneapolis, Minnesota

Calogera M. Simonaro - Molecular mechanisms underlying joint and bone disease in the mucopolysaccharidoses (MPS).
Mount Sinai School of Medicine, New York, New York

Michael L. West - Withdrawal of enzyme replacement therapy in fabry disease: indirect evidence of treatment benefit?
Dalhousie University, Halifax, Nova Scotia, CANADA

William R. Wilcox - Females with x-linked Fabry disease frequently have significant organ involvement.
Cedars-Sinai Medical Center, Los Angeles, California

Tim Wood - Molecular and biochemical studies of MPS II patients and families.
Greenwood Genetic Center, Greenwood, South Carolina

Sandra Yang - Treat type I Gaucher disease with Miglustat - experience with two patients.
Children's National Medical Center, Washington, D.C.