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This Symposium is the second in the series of Continuing Medical Education activities organized by Serono Symposia International Foundation for the European Phenylketonuria Group (EPG). Present and future efforts on phenylketonuria (PKU) treatment will be discussed in this event particularly with regard to new pharmacological approaches, such as enzyme substitution and cofactor administration. The importance of patient management will also be highlighted, both in PKU as well as in tetrahydrobiopterin deficient patients. Furthermore, the workshop sessions will offer the possibility to discuss knowledge and experiences on six major aspects of PKU management. The final aim of this Symposium is the improvement of patient management based on sharing the most updated knowledge.
After attending this symposium, the participants will have an updated knowledge on:
Serono Symposia International Foundation (www.seronosymposia.org) will submit this program “2nd EPG Symposium - Advances and Challenges in PKU” (Munich, Germany - January 22-23, 2010) for accreditation by the European Accreditation Council for Continuing Medical Education (EACCME), the Royal College of Physicians and the Italian Ministry of Health.
NB. All Serono Symposia International Foundation programs are organized solely to promote the exchange and dissemination of scientific and medical information. No forms of promotional activities are permitted. There may be presentations discussing investigational uses of various products. These views are the responsibility of the named speakers and do not represent an endorsement or recommendation on the part of Serono Symposia International Foundation.
Friday, January 22, 2010
Session I: Advances in PKU research
Chairmen: Charles R. Scriver (Canada); Francjan J. van Spronsen (The Netherlands)
Phenylalanine Ammonia Lyase (PAL) Enzyme substitution therapy for Phenylketonuria (PKU) and clinical trials
Nicola Longo, USA
Genotype-phenotype correlation in PKU and the contribution of mutation molecular pathology
Steven F. Dobrowolski, USA
Mechanism of BH4-responsiveness in PKU
Ania C. Muntau, Germany
Parallel Workshops
WORKSHOP 1: Sapropterin (BH4) and diet in the management of PKU
Chairmen: Amaya Bélanger-Quintana, Spain - Friedrich K. Trefz, Germany
WORKSHOP 2: Assessment of quality of life in PKU: How should we do this?
Chairmen: Annet M. Bosch, The Netherlands - Mübeccel Demirkol, Turkey
WORKSHOP 3: Neuropsychological evaluation of PKU patients: What are the standards?
Chairmen: Reinhold Feldmann, Germany - Francjan J. Van Spronsen, Netherlands
WORKSHOP 4: PKU in adult life: Risks and assessment of treatment efficiency
Chairmen: Harold W. de Valk, The Netherlands - François Feillet, France
WORKSHOP 5: Nutritional follow-up of PKU patients
Chairmen: Marcello Giovannini, Italy - Anita MacDonald, UK
WORKSHOP 6: Diagnosis of BH4 responsive PKU patients
Chairmen: Nenad Blau, Switzerland - Barbara Burton, USA
Saturday, January 23, 2010
Session II: Patients Management
Chairmen: Amaya Bèlanger-Quintana (Spain); Mübeccel Demirkol (Turkey)
MRS in the management of PKU
Roland Kreis, Switzerland
BH4 deficiencies: Diagnosis, treatment and follow-up
Alberto Burlina, Italy
To treat or not to treat mild hyperphenylalaninaemia
Francjan J. Van Spronsen, Netherlands
Selected Oral Communications
Chairmen: François Feillet (France); Anita MacDonald (UK)
Direct in-well activity measurement of phenylalanine hydroxylase reveals positive cooperativity for the binding of BH4
Michael Staudigl, Germany
Pharmacological chaperones for the aromatic amino acid hydroxylases
Beat Thöny, Switzerland
Long-chain polyunsaturated fatty acid status in phenylketonuric patients treated with tetrahydrobiopterin
Maria Antonia Vilaseca, Spain
Phenylketonuria: Correlations between lumbar bone mineral density and vitamin and mineral blood concentrations
Martijn de Groot, Netherlands
Visual evoked potentials and auditory mismatch negativity in children with earlytreated PKU. Effects of concurrent and lifetime Phe levels
Leo M.J. de Sonneville, Netherlands
Cost and availability of special dietary products for PKU in Europe
Margreet van Rijn, The Netherlands
Treatment of six maternal PKU pregnancies with BH4 and a phenylalanine restricted diet
Richard Koch, USA
The KAMPER Registry: A long term observational study of patients with hyperphenylalaninemia treated with sapropterin dihydrochloride
François Feillet, France
Session III: Meet Pioneers in PKU
Round table
Moderator: John Illman (UK)
Milan Blaskovics, USA
David P. Brenton, UK
Forrester Cockburn, UK
Flemming Güttler, Denmark
Richard Koch, USA
Harvey L. Levy, USA
Isabel Smith, UK
Friedrich K. Trefz, Germany
ASBJØRN FØLLING LECTURE “GIANTS IN PKU”
The Folling Award Lecture: Some Things Considered
Charles R. Scriver, Canada
Amaya Bélanger Quintana
Unidad de Enfermedades Metabólicas
Servicio de Pediatría
Hospital Ramón y Caja
Madrid, Spain
Nenad Blau
Division of Clinical Chemistry and Biochemistry
University Children's Hospital
Zürich, Switzerland
Mübeccel Demirkol
Children's Hospital
Istanbul Faculty of Medicine
Istanbul University
Istanbul, Turkey
François Feillet
Centre de Référence des Maladies Héréditaires du Métabolisme
Hôpital d’Enfants, CHU Brabois
Vandoeuvre les Nancy, France
Marcello Giovannini
Department of Pediatrics
San Paolo Hospital
University of Milan
Milan, Italy
Anita MacDonald
Dietetic Department
The Children's Hospital
Birmingham, UK
Friedrich K. Trefz
Children’s Hospital
Kreis Kliniken Reutlingen GmbH
Klinikum Am Steinberg
School of Medicine University of Tuebingen
Reutlingen, Germany
Francjan J. van Spronsen
Beatrix Childrens Hospital
University Medical Center of Groningen
Groningen, The Netherlands
Serono Symposia International Foundation adheres to guidelines of the European Accreditation Council for Continuing Medical Education (EACCME) and all other professional organizations, as applicable, which state that programs awarding continuing education credits must be balanced, independent, objective, and scientifically rigorous. Investigative and other uses for pharmaceutical agents, medical devices, and other products (other than those uses indicated in approved product labeling/package insert for the product) may be presented in the program (which may reflect clinical experience, the professional literature or other clinical sources known to the presenter). We ask all presenters to provide participants with information about relationships with pharmaceutical or medical equipment companies that may have relevance to their lectures. This policy is not intended to exclude faculty who have relationships with such companies; it is only intended to inform participants of any potential conflicts so participants may form their own judgments, based on full disclosure of the facts. Further, all opinions and recommendations presented during the program and all program-related materials neither imply an endorsement, nor a recommendation, on the part of Serono Symposia International Foundation. All presentations solely represent the independent views of the presenters/authors.
The following faculty provided information regarding significant commercial relationships and/or discussions of investigational or non-EMEA/FDA approved (off-label) uses of drugs:
Jan Alm: Declared to be a member of Merck Serono SA Advisory Board and receipt of consulting fees from Merck Serono SA.
Amaya Bélanger-Quintana: Declared to be a member of Merck Serono SA Advisory Board and receipt of consulting fees from Merck Serono SA.
Milan E. Blaskovics: Declared no potential conflict of interest.
Nenad Blau: Declared receipt of grants and contracts from Merck Serono SA, Switzerland.
Olaf Bodamer: Declared receipt of honoraria from Merck Serono SA.
Annet M. Bosch: Declared receipt of grants and contracts from SHS International and to be member of Merck Serono Advisory Board.
David P. Brenton: Declared no potential conflict of interest.
Alberto Burlina: Declared receipt of consulting fees from Merck Serono SA.
Barbara Burton: Declared receipt of grants, contracts honoraria or consultation fees from BioMarin Pharmaceutical and declared to participate in BioMarin Pharmaceutical Speaker’s Bureau.
Agnes Champigneulle: Declared to be an employee of Merck Serono SA.
Forrester Cockburn: Declared no potential conflict of interest.
Amy Cole: Declared receipt of honoraria from Merck Serono SA, Geneva.
Harold W. de Valk: Declared no potential conflict of interest.
Mübeccel Demirkol: Declared receipt of honoraria or consultation fees from Merck Serono SA, Geneva.
Steven F. Dobrowolski; Declared receipt of grants and contracts from National Institute of Health of the United States.
François Feillet; Declared to be a member of Merck Serono SA Advisory Board and the principal investigator for the KAMPER registry.
Reinhold Feldmann: Declared no potential conflict of interest.
Ivar Følling: Declared no potential conflict of interest.
Georg Frauendienst-Egger: Declared no potential conflict of interest.
Marcello Giovannini: Declared to be a member of PKU Advisory Board.
Richard Koch: Declared no potential conflict of interest.
Roland Kreis: Declared no potential conflict of interest.
Nicola Longo: Declared receipt of grants, contracts honoraria or consultation fees from BioMarin Pharmaceutical Inc.
Anita MacDonald: Declared receipt of grants and contracts from SHS International and Vitaflo and declared to be a member of Merck Serono Advisory Board.
Ania C. Muntau: Declared receipt of research grants for scientific work from SHS GmbH, Germany and from Merck Serono GmbH, Darmstadt. Declared to be a member of PKU Scientific Advisory Board and receipt of
honoraria from Merck Serono SA.
Charles R. Scriver: Declared receipt of grants and contracts from BioMarin, USA.
Isabel Smith: Declared no potential conflict of interest.
Friedrich K. Trefz: Declared receipt of honoraria or consultation fees from Merck Serono SA and to be a member of Merck Serono Advisory Board.
Francjan J. van Spronsen: Declared to be a member of Merck Serono Scientific Advisory Board for Phenylketonuria.
The following faculty have provided no information regarding significant relationship with commercial supporters and/or discussion of investigational or non-EMEA/FDA approved (off-label) uses of drugs as of January 13, 2010.
Flemming Güttler
Harvey L. Levy
Nenad Blau
Division of Clinical Chemistry and Biochemistry
University Children's Hospital
Zürich, Switzerland
Anita MacDonald
Dietetic Department
The Children's Hospital
Birmingham, UK
Francjan J. van Spronsen
Beatrix Childrens Hospital
University Medical Center of Groningen
Groningen, The Netherlands
