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