Program of the Conference
Friday 2 July
09.00 – 09.05 hrs. Chairman Mr. R. House, AMDA (USA)
09.05 – 09.10 hrs. Opening of the International Pompe Conference
Mr. Y.S. Poortman, VSN (The Netherlands)
Research in Enzyme Replacement Therapy
09.10 – 09.40 hrs. “Clinical variability in Pompe’s disease”
Dr. M.C.B. Loonen, Rotterdam (The Netherlands)
09.40 – 10.10 hrs. “Epidemiological studies in glycogen storage disease type II
Dr. M.G.E.M. Ausems, University Medical Centre Utrecht (The Netherlands)
10.10 – 10.40 hrs. “Early detection and diagnosis of Pompe”
Prof. J.J. Hopwood, Women’s & Children’s Hospital Adelaide (Australia)
10.40 – 11.00 hrs. Coffee break
11.00 – 11.30 hrs. “Pompe disease in historical perspective”
Dr. A.J.J. Reuser, Erasmus University Rotterdam (The Netherlands)
11.30 – 12.30 hrs. “A Transgenic Approach to Enzyme Replacement Therapy in Pompe Patients”
Mr. A. Vos, MSc, Pharming/Genzyme LLC (The Netherlands), Mr. J. Dalle, MSc, Pharming NV (Belgium), Mr. A. Curtis, MBA, Genzyme Corp. (USA)
12.30 – 14.00 hrs. Lunch
14.00 -14.30 hrs. “Therapy for GSD-II: Enzyme based therapeutics and novel gene therapy approaches”
Dr. A. Amalfitano, Duke University Medical Centre (USA)
14.30 -15.00 hrs “The Challenges of rhGAA Manufacture”
Dr. B. Devlin, Synpac Pharmaceuticals (UK)
15.00 -15.30 hrs. Teabreak
PATIENT INITIATIVE AND ETHICS
15.30 – 16.00 hrs “From patient initiative to treatment”
Ms. M. Schoneveld van der Linde, MA, VSN (The Netherlands)
16.00 – 16.30 hrs. “Knowledge is power”
Dr. K. O’Donnell, AGSD (UK)
16.30 – 17.00 hrs. “How to do good and to prevent harm?”
Dr. H.J.M.A. van den Boer- van den Berg, Academic Hospital Rotterdam
(The Netherlands)
Due to circumstances Ms. Van den Boer-van den Berg is not able to attend the conference.
19.00 hrs. Dinner Tulip Inn, Naarden
Saturday 3 July
09.30 – 09.35 hrs. Chairperson Ms. R. Broekgaarden, VSN (The Netherlands)
Pompe Groups; Working on a National Level
09.35 – 10.05 hrs. “Forming a national Pompe group our experience”
Ms. M. House, AMDA (USA)
10.05 – 10.35 hrs. “The International Pompe Association; position, objectives, policy and activities”
Mr. Y.S. Poortman, VSN (The Netherlands)
10.35 – 11.00 hrs. Coffee break
The Need for, and Nature of, Patient Involvement: the International Pompe Association (IPA)
11.00 – 12.30 hrs. “Developments in the area of Pompe’s disease”
Mr. H. van Veenendaal, VSN (The Netherlands)
“Funding Treatment before the Government Subsidy Approvals”
Mr. R. Morrison, MDA (Australia)
“Legal procedures regarding the International Pompe Association”
Mr. H. Erny, SHG Glykogenose Deutschland e.V. (Germany)
“The strength of networking”
Mr. R.H. House, AMDA (USA)
“The importance of good information”
Dr. K. O’Donnell, AGSD (UK)
Round Table Discussion
12.30 – 14.00 hrs. Lunch
14.00 – 16.00 hrs. Closed session for patient-representatives
Round Table Discussion
after 16.00 hrs. Evening program
Sunday 4 July
09.00 – 09.05 hrs. Chairperson Mr. T. Schaller, SHG Glykogenose Deutschland (Germany)
09.05 – 10.35 hrs. Closed session for patient-representatives
“Agreements and actions of the International Pompe Association”
Mr. Y.S. Poortman, VSN (The Netherlands)
10.35 – 10.50 hrs. Coffee break
10.50 – 11.30 hrs. “Enzyme therapy for Pompe’s disease”
Dr. A.T. van der Ploeg, Sophia Children’s Hospital Rotterdam (The Netherlands)
11.30 – 12.30 hrs. Lunch
13.00 hrs. Departure