AMDA
Projects
1996--First Scientific
Symposium
In March 1996 AMDA
sponsored and funded the first scientific symposium
dealing exclusively with Pompe's disease. It was attended
by leading investigators from around the world. This
conference played a major role in accelerating research
in this field. Until this symposium the medical and
scientific community had minimal contact and there was a
lack of communication among the investigators. New
collaborative efforts were forged at this meeting.
Attending the meeting in San Antonio, Texas, were:
Y.T. Chen, M.D., Ph.D.
Johan Van Hove, M.D., Ph.D.
Andrew Balber, Ph.D.
Blythe Devlin, Ph.D.
Duke University Medical Center
Durham, North Carolina
Arnold J.J. Reuser, Ph.D.
Ans T. van der Ploeg, M.D., Ph.D.
Erasmus University & Sophia's Children Hospital
Rotterdam, the Netherlands
Rochelle Hirschhorn, M.D.
Frank Martiniuk, Ph.D.
Maryann Hui, M.D., Ph.D.
New York University Medical Center
New York, New York
James Sidbury, M.D.
Paul H. Plotz, M.D.
Nina Raben, M.D., Ph.D.
Joseph Zaretsky, M.D., Ph.D.
Ralph Nichols, Ph.D.
National Institutes of Health
Bethesda, Maryland
Alfred E. Slonim,
North Shore University Hospital
Manhasset, New York
Tateki Kikuchi, Ph.D.
Seiichi Tsujino, M.D.
National Institute of Neuroscience
Tokyo, Japan
Clare Beesley, Ph.D.
Harefield Hospital
Middlesex, United Kingdom
Olaf Bodamer, M.D.
Institute of Child Health
London, England
Peter Healy, B.V.Sc., Ph.D.
Elizabeth MacArthur Agricultural Institute
Camden, Australia
Richard Bertin, Ph.D., Director,
Grant Program
Orphan Products Development, FDA
Rockville, Maryland
Randall H. House, President of AMDA,
officiated.
1997--Second Scientific Symposium
AMDA sponsored the
second scientific conference in June 1997.
The three day seminar was held in San Antonio, Texas and
was sponsored in part by Pharming B.V. Following
is a list of investigators present at the conference:
Arnold J.J. Reuser, Ph.D.
Ans T. van der Ploeg, M.D., Ph.D.
Erasmus University & Sophia's Children
Hospital Rotterdam, the Netherlands
Y.T. Chen, M.D., Ph.D.
Andy Amalfitano, D.O., Ph.D.
Duke University Medical Center
Durham, North Carolina
Tateki, Kikuchi, Ph.D.
Seiichi Tsujino, M.D.
National Institute of Neuroscience
Tokyo, Japan
Alfred E. Slonim, M.D.
North Shore University Hospital
Manhasset, New York
Rochelle Hirschhorn, M.D.
New York University Medical Center
New York, New York
Paul H. Plotz, M.D.
Nina Raben, M.D., Ph.D.
Bo Yan, Ph.D.
National Institutes of Health
Bethesda, Maryland
Paul D. Kessler, M.D.
Barry J. Byrne, M.D., Ph.D.
Johns Hopkins University
Baltimore, Maryland
J. Clarke McIntosh, M.D.
Ruth and Billy Graham Children's Health Center
Asheville, North Carolina
Livia Poenaru, M.D., Ph.D.
Rene Descartes University
Paris, France
Marc P. Nicolino, M.D.
Debrousse Hospital
Lyon, France
Cesare Danesino, M.D.
University of Pavia
Pavia, Italy
Ching-Yuang Lin, M.D., Ph.D.
Jeng-Jer Shieh, Ph.D.
Veteran General Hospital-Taipei and
National Yang Ming University
Taipei, Taiwan, R.O.C.
Patricia Robuck, M.P. H., Grant Program Director
Ronda Balham, O.D.
Orphan Products Development, FDA
Rockville, Maryland
Gerben Moolhuizen, M.B.A., Pompe
Project Director
Emile van Corven, Ph.D.
Pharming B.V.
Leiden, the Netherlands
Randall H. House, President of AMDA, officiated.
1998--AMDA Third International
Workshop
The Third International Workshop
of the AMDA, Acid Maltase Deficiency Association, was
held on December 3-5, 1998, at the National Institutes of
Health (NIH) in Bethesda, Maryland. Sixty-five
international scientists and physicians that are doing
research in AMD, acid maltase deficiency, or in closely
related areas attended the conference.
Presentations were made on enzyme
replacement therapy, gene therapy, and other approaches
to treatment for AMD. Research information on other
metabolic disorders which may be relevant to AMD was also
presented.
This conference was sponsored by the
following Intramural Research Programs of the NIH:
National Institute of Arthritis
and Musculoskeletal and Skin Diseases (NIAMS)
National Heart, Lung and Blood
Institute (NHLBI)
National Institute of Child
Health and Human Development (NICHD)
National Institute of Diabetes
and Digestive and Kidney Disease (NIDDK)
National Institute of Mental
Health (NIMH)
National Institute of
Neurological Disorders and Stroke (NINDS)
Office of Rare Disease Research
(ORDR)
Other Contributors:
Funding by AMDA
AMDA has funded or
procured funds totaling over $4,900,000 for
projects directed toward the treatment and cure of Acid
Maltase Deficiency. Following is a partial list of grant
recipients:
Duke University Medical Center
Y.T. Chen, M.D., Ph.D., & Johan Van Hove,
M.D., Ph.D.
Purchase of FPLC Protein Purification Equipment
(automated FPLC work station, motor valve,
chromatography refrigerator and accessories)
New York University Medical
Center
Rochelle Hirschhorn, M.D.
&
Hebrew University of Jerusalem, Israel
Shimon Gatt, M.D., Ph.D.
Development of an Intracellular Specific Assay to
Diagnose GAA Deficiency by Targeting the Fluorescent
Substrates to the Lysosomes
North Shore University Hospital
Alfred E. Slonim, M.D.
Study on Management of Adult Onset Acid Maltase
Deficiency
Through the Use of Diet and Ephedrine
Erasmus University,
Rotterdam, The Netherlands
Arnold J.J. Reuser, Ph.D.
Grant for Research Aimed at the Development of a
Therapy for Acid Maltase Deficiency
AMDA has also been
instrumental in procuring funds for the following:
New York University Medical
Center
Frank Martiniuk, Ph.D.
Grant for Research of Biochemical and Molecular Analysis
of Activator Protein for Acid Alpha-Glucosidase
Research Directed by
Pharming B.V. , Leiden, the Netherlands
in conjunction with:
Erasmus University, Arnold J.J. Reuser, Ph.D.
Sophia's Children Hospital, Ans T. van der Ploeg, M.D.,
Ph.D.
Rotterdam, the Netherlands
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