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Family Program

Preliminary program - subject to changes

14. International Symposium on MPS and Related Diseases

Thursday, 14th July 2016
15:00 - 15:30Opening Ceremony
15:30 - 16:00Opening Lecture
Chair: Kurt Ullrich
Latest Understanding in the Biology of the
Lysosomal Network Volkmar Gieselmann, Germany
16:00 - 18:00
Putting the Patient at the Center of Care
Chair: Christine Lavery and Kurt Ullrich
16:00 - 16:30The Scientific Challenges to Delivering New Therapies
for the MPS Diseases Brian Bigger, UK
16:30 - 17:00Clinical Trial and Access to New Therapies
for MPS Diseases Emil D. Kakkis, USA
17:00 - 17:30Treatment and Monitoring of MPS Patients
with Multisystemic Involvement Roberto Giugliani, Brazil
17:30 - 18:00Empowering Patients and the Future Role
of Patient Organisations Georg Schetter, Germany
18:00 - 20:00Welcome Reception

Friday, 15th July 2016
08:45 - 10:15Clinical Management
Chair: Bryan Winchester and Joseph Muenzer
08:45 - 09:05How to Predict the Clinical Phenotype in MPS?
Roberto Giugliani, Brazil
09:05 - 09:30Neurodevelopment in MPS Syndromes, Natural History
and Treatment Outcomes Maria Luisa Escolar, USA
09:30 - 09:50Neurological Problems in MPS: Clinical Manifestations
and Treatment Maurizio Scarpa, Germany
09:50 - 10:15The Natural History of Cognitive, Behavioural and
Neuroimaging Characteristics of MPS III Elsa Shapiro, USA
10:15 - 10:45Coffee Break
10:45 - 12:15
New Therapies and Clinical Trials I
Chair: Christine Lavery and Nicole Muschol
10:45 - 10:55Understanding Gene Therapy Patricia Dickson, USA
10:55 - 11:05Intracerebral Gene Therapy for MPS III A Samantha Parker, France
11:05 - 11:15Intrathecal Gene Therapy for MPS III A/B Fátima Bosch-Tubert, Spain
11:15 - 11:25Intravenous Gene Therapy on MPS III A/B Haiyan Fu, USA
11:25 - 11:35Stem Cell Gene Therapy for MPS III A/B and MPS II Brian Bigger, UK
11:35 - 11:45Intracerebral Gene Therapy for MPS III C Claire O’Leary, UK
11:45 - 11:55Intravenous Gene Therapy for MPS VI Generoso Andria, Italy
11:55 - 12:15Discussion
12:15 - 14:00Lunch Break
14:00 - 15:30Clinical Management
Chair: Maria Luisa Escolar and Carla Hollack
14:00 - 14:25Sleep Disorders in MPS: Changes of the Circadian Rhythm Stewart Rust, UK
14:25 - 14:40Pregnancy in MPS Patients Susanne Kircher, Austria
14:40 - 15:05The Experiences of Women with MPS during Pregnancy
and Beyond Deborah Cavell, UK
15:05 - 15:30MPS Transition- Ensuring Safe Care in Adulthood
Christian Hendriksz, UK
15:30 - 16:00Coffee Break
16:00 - 17:40
New Therapies and Clinical Trials II
Chair: Michael Beck and Anne-Sophie Lapointe
16:00 - 16:20Intrathecal ERT for MPS II and III A Simon Jones, UK
16:20 - 16:40Intracerebroventricular ERT for MPS III B Nicole Muschol, Germany
16:40 - 17:00ERT (not intrathecal) Trial for MPS III B Maria Luisa Escolar, USA
17:00 - 17:20Fusion Protein Therapy for MPS I and II Roberto Giugliani, Brazil
17:20 - 17:40Trial with Small Molecule Drug for MPS I n/m Christina Lampe, Germany
18:00 - 20:00Remembrance Ceremony

Saturday, 16. Juli 2016
08:45 - 10:15  Pitfalls and Advances in Therapy
Chair: Michael Beck and Frits Wijburg
08:45 - 09:10 When to Start and to Stop ERT in MPS? Simon Jones, UK
09:10 - 09:30How to Manage Immunological Reactions Paul Harmatz, USA
09:30 - 09:55Transplantation Experiences in MPS Diseases:
Success, Limitations and Future Considerations Paul J. Orchard, USA
09:55 - 10:15Pentose-Sulfate: New Mechanistic Insights
as a Treatment for MPS Calogera Simonaro, USA
10:15 - 10:45Coffee Break
10:45 - 12:15Advanced Nursing Care
Chair: Christine Lampe and Hanka Dekker
10:45 - 11:00Palliative Care and Ethical Aspects Anne-Sophie Lapointe, France
11:00 - 11:15Can we Ease the Disease Burden on the Families? Veronica Hübinette, Sweden
11:15 - 11:35Pain Management in Mucopolysaccharidosis Chiara di Pede, Italy
11:35 - 11:55Gastrostomy Feeding and MPS Jens Berrang, Germany
11:55 - 12:15Sleep Disturbances Simon Jones, Stewart Rust, UK
12:15 - 13:45Lunch Break
13:45 - 15:15
The Role of Complementary Therapies
Chair: Lorenz Grigull and Marija Joldic
13:45 - 14:05Dysphagia as a Therapeutic Support Sabine Burghardt-Oehm, Germany
14:05 - 14:25Physiotherapy Christine Wurlitzer, Germany
14:25 - 14:45Respiratory Therapy with Music and iPad Ruud van der Wel, The Netherlands
14:45 - 15:05Craniosacral Therapy Ulrike Kaser, Austria
15:05 - 15:15Discussion
15:15 - 16:00Coffee Break
16:00 - 17:30
Practical Learning Workshops on Complementary Therapies
16:00 - 17:30Workshop on Dysphagia Sabine Burghardt-Oehm, Germany
16:00 - 17:30Workshop on Important Aspects in Physiotherapy
Christine Wurlitzer, Germany
16:00 - 17:30Workshop on Music Therapy Ruud van der Wel, The Netherlands
16:00 - 17:30
Adult Perspectives of Living with MPS
Chair: Michaela Weigl and Susanne Kircher
16:00 - 17:30Eric Ma, MPS VI, Hongkong
Deise Zanin, MPS I, Brasilien
Alexia Zimmer MPS VI, Germany
Maria Prähofer, MPS IV, Austria
Jibreel Arshad, MPV IV, UK
Jason Madison, MPS II USA
19:30 - 22:30Conference Dinner

Sunday, 17. Juli 2016
08:30 - 10:15MPS Adults (Hall Schumann): Sports and physiotherapy with MPS
Torge Wittke and Christine Wurlitzer, Germany
08:30 - 10:15Learnings from Recent Advances in Therapy of LSDs
Chair: Andres Klein and Nicole Muschol
08:30 - 08:55Delivery of Enzyme - Fusion Proteins to the Brain Ruben Boado, USA
08:55 - 09:25Intrathecal ERT in MPS Patricia Dickson, USA
09:25 - 09:55Gene Therapy for Neurologic Manifestation of MPS Joseph Muenzer, USA
09:55 - 10:15Corneal Transplantation in MPS Susanne Pitz, Germany
10:15 - 10:45Coffee Break
10:45 - 12:15MPS Clinical Management - Best Care
Chair: Emil Kakkis and Kurt Ullrich
10:45 - 11:10 The Changing Face of Anaesthesia for the Mucopolysaccharidoses
Robert Walker, UK
11:10 - 11:35Orthopaedic Management of Thoracic Spine Disease
Neil Oxborrow, UK
11:35 - 11:55Hip Disease:Natural History and Possible Indications for Orthopaedic Interventions Andrea Borgo, Italy
11:55 - 12:15The Role of Clinical Guidelines in Access to High Cost Therapies 
Carla Hollak, The Netherlands
12:15 - 12:45The Future: Combination Therapies?
Frits Wijburg, The Netherlands
12:45 - 13:00
Presentation for 2018 Patricia Dickson, USA /