Saturday 30. June 2012
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| 09:00 |
Session 5 - scientific & patient program
Novel approaches for treatment and early diagnosis
Chairs: George Ruiter & John Hopwood |
09:00
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Intrathecal enzyme therapy for MPS II and III
Joseph Muenzer, USA
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09:20
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Genistein: results of clinical trials
Frits Wijburg, the Netherlands
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09:40
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Genistein: animal studies and a new trial
Brian Bigger, United Kingdom
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10:00
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Animal models and innovative therapies for brain disease
Kim Hemsley, Australia
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10:20
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General discussion
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10:30
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Coffee break
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11:00
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Session 6 - scientific program
Novel approaches for treatment and early diagnosis challenges and solutions
Chairs: Arnold Reuser & Vassili Valayannopoulos
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11:00
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Gene Therapy for Sanfilippo syndrome
Jean Michel Heard, France
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11:20
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Gene therapy for MPS VI
Alberto Auricchio, Italy
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11:40
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Gene therapy by transfecting autologous hematopoietic stem cells
Alessandra Biffi, Italy
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Oral presentations |
12:00
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A single systemic rAAV9-hNAGLU gene delivery: normalized survival and permanent restoration of NAGLU activity in the CNS, PNS and somatic tissues in MPS IIIB mice
Haiyan Fu, USA |
12:10
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Lentiviral SGSH expression from the human CD11b promoter in transplanted haematopoietic stem cells fully corrects behaviour and neuropathology of Mucopolysaccharidosis IIIA mice
Alexander Langford-Smith, United Kingdom
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12:20
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Heparan Sulphate Inhibits CXCL12-mediated Hematopoietic Cell Migration and Engraftment in a Mouse Model of Mucopolysaccharidosis I
Brian Bigger, United Kingdom
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12:30
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Lunch
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12:30
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Lunch symposium - Shire
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13:30
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Session 7 - scientific & patient program
Drug pricing and access to treatment
Chairs: Ans van de Ploeg & Alasdair Parker
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13:30
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Orphan drug development: successes and challenges
Bert Leufkens, the Netherlands
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13:50
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Perspectives of patients and families
Christine Lavery, United Kingdom
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14:10
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The doctor’s perspective
Carla Hollak, the Netherlands
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14:30
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Perspective of regulating authorities
Wim Goettsch, the Netherlands
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14:50
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Cost effectiveness and orphan drugs
Michael Schlander, Germany
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15:10
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General discussion
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15:30
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Tea break
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16:00
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Session 8 - scientific & patient program
Updates on treatment outcomes
Chairs: Maurizio Scarpa & David Begley
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16:00
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MPS I
Roberto Giugliani, Brazil
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16:15
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MPS II
Michael Beck, Germany
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16:30
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MPS IV
Chris Hendriksz, United Kingdom
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16:45
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MPS VI
Vassili Valayannopoulos, France
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Oral presentations |
| 17:00 |
Anti-idursulfase Antibody and the Efficacy of Enzyme Replacement Therapy in Mucopolysaccharidosis Type II Patients
Akemi Tanaka, Japan
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| 17:10 |
Long-term Experience with Enzyme Replacement Therapy (ERT) in MPS II Patients with a Severe (Neuronopathic) Phenotype: an International Case Series
Christina Lampe, Germany
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17:20
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International multicenter study to identify predictors of long term outcome of Hurler syndrome patients after successful hematopoietic stem cell transplantation
Jaap Jan Boelens, the Netherlands
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17:30
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Lysosomal storage, neurodegeneration and brain inflammation in the mouse model of MPS III type C
Alexey Pshezhetsky, Canada
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17:40
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Drinks & snacks & poster walks
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19:00
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Congress dinner
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