2008 LYSOSOMAL DISEASES AND THE BRAIN CONFERENCE
a conference hosted by The Children's Gaucher
Research Fund
2008 CONFERENCE FEATURED SPEAKERS
Speakers are listed in alphabetical order, by last name.
Chief, CBMP
National Institutes of Health, Bethesda, Maryland
Email: juan@helix.nih.gov
Talk: “Molecular Mechanisms of Lysosome Biogenesis”
Roscoe O. Brady M.D.
Scientiest Emeritus - National Institute of Neurological Disorders and Stroke
National Institutes of Health, Bethesda, Maryland
Email: rb57v@nih.gov
Talk: “The Future of Gaucher disease research”
Seng H. Cheng Ph.D.
Group Vice President, Genetic Diseases Science
Genzyme Corporation, Framingham, Massachusetts
Email: seng.cheng@genzyme.com
Talk: “Prospects for Treating the CNS Disease in LSDs”
Jonathon Cooper Ph.D.
Reader in Experimental Neuropathology
Institute of Psychiatry, King's College London
Pediatric Storage Disorders Lab, Department of Neuroscience,
King's College, London, United Kingdom
Email: j.cooper@iop.kcl.ac.uk
Talk: “Common themes in Batten disease pathogenesis”
Ana Maria Cuervo M.D. Ph.D
Associate Professor - Department of Anatomy and Structural Biology
Albert Einstein College of Medicine
Bronx, New York
Email: amcuervo@aecom.yu.edu
Talk: “Autophagy in the pathophysiology of the brain”
Alessandra d'Azzo Ph.D.
Member and Endowed Chair
St. Jude Children’s Research Hospital, Department of Genetics
Memphis, Tennessee
Email: sandra.dazzo@stjude.org
Talk: “GM1-ganglioside as apoptotic signal in ER- and mitochondria-mediated cell death in neurodegenerative GM1 gangliosidosis”
Tony Futerman Ph.D.
Professor, Department of Biological Chemistry
Weizmann Institute of Science
Rehovot, Israel
Email: tony.futerman@weizmann.ac.il
Talk: “Overview of pathological mechanisms in sphingolipid storage disease of the brain”
Volkmar Gieselmann M.D.
Professor , Institute of Physiological Chemistry
University of Bonn
Bonn, Germany
Email: gieselmann@institut.physiochem.uni-bonn.de
Talk: “Therapeutic trials and pathogenetic studies in an animal model of metachromatic leukodystrophy”
Gregory A. Grabowski M.D.
Professor and Director, Division of Human Genetics
The A. Graeme Mitchell Chair in Human Genetics
University of Cincinnati, Children’s Hospital Research Foundation of Cincinnati
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio
Email : greg.grabowski@cchmcorg
Talk: “Insights into the function of saposins and mutant glucocerebrosidases in the CNS”
Makoto Ito, Ph. D.
Professor
Department of Bioscience and Biotechnology and Bio-Architecture Center
Kyushu University, Japan
Email: makotoi@agr.kyushu-u.ac.jp
Talk: "Novel metabolic pathway of glucosylceramide involving Klotho-related protein: Insights into Gaucher disease"
Gerrit van Meer Ph.D.
Professor
Membrane Enzymology, Bijvoet Center and Institute of Biomembranes,
Utrecht University
The Netherlands
Email: g.vanmeer@uu.nl
Talk: “Fate and function of glucosylceramide in mammalian cells”
William M. Pardridge M.D. (Bill)
Professor of Medicine
University of California at Los Angeles
Los Angeles, California
Email: wpardridge@mednet.ucla.edu
Talk: “Re-Engineering Lysosomal Enzymes for Intravenous Delivery to the Human Brain”
Fran Platt, Ph.D.
Department of Pharmacology
University of Oxford
Oxford, United Kingdom
Email: frances.platt@pharm.ox.ac.uk
Talk: “New therapeutic approaches for lysosomal disorders of the brain”
Raphael Schiffmann M.D.
Chief, Clinical Investigations, DMNB
National Institutes of Health
Bethesda, Maryland
Email: schiffmr@ninds.nih.gov
Talk: “Therapy – what's on the horizon?”
Ellen Sidransky, M.D.
Chief, Section on Molecular Neurogenetics
MGB, National Human Genome Research Institute
National Institutes of Health
Email: sidranse@mail.nih.gov
Talk: "Gaucher disease and Parkinsonism"
Einat Vitner Ms.c
Department of Biological Chemistry
Weizmann Institute of Science
Rehovot , Israel
Email: einat.vitner@weizmann.ac.il
Talk: “The role of defective calcium-homeostasis in the neuropathology of type 2 and 3 Gaucher disease”
Col. Kondi Wong M.D. USAF MC
Chief, Division of Neuropathology
Wilford Hall Medical Center
Lackland, AFB
San Antonio, Texas
Email: kondi.wong@lackland.af.mil
Talk: “Overview of pathological mechanisms in sphingolipid storage disease of the brain”
Brandon A. Wustman Ph.D.
Principal Scientist
Amicus Therapeutics
Cranbury, New Jersey
Email: BWustman@amicustherapeutics.com
Talk: “Pharmacological chaperones as a potential therapy for lysosomal disorders of the brain”
Yildiz Yildiz M.D.
University of Bonn, Internal Medicine I 4
Bonn , Germany
Email: yildiz.yildiz@ukb.uni-bonn.de
Talk: “Glucosylcerebrosid accumulation in gba2 knockout mouse”
Contact Us: Tel: 916.797.3700 • Fax: 916.797.3707 • e-mail:
research@childrensgaucher.org
P.O. Box 2123 • Granite Bay, California 95746-2123