International Workshop

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International Workshop Alport Syndrome September, 25-27, 2015 Göttingen

Welcome Dear friends, the last workshop in Oxford has shown us how valuable it is to work at the international level in a rare serious disease such as Alport syndrome. The 2015 workshop in Göttingen wants to continue this spirit of Oxford. At a historic site in Göttingen, the Old Gauss Observatory, we will cover clinical, genetic, and basic science aspects of the disease. The workshop is determined to develop strategies and collaborations for the future, with the overall aim of discovering much needed new treatments to prolong kidney function and to prevent hearing loss. We cordially invite you as stakeholders from diverse communities - patient families, physicians, geneticists, researchers, Pharma, and funding organizations to come together to hear, learn and discuss about Alport syndrome. The Scientific Committee will award the best presentations by young researchers dedicated to research in Alport s syndrome. We feel honored to confirm that KDIGO is committed to working with us on this conference. Prof. Oliver Gross congress president Page 1

Scientific Committee Professor Colin Baigent, MRC Scientist, Hon Consultant in Public Health, Oxford, UK Professor Frances Flinter, Guy s Hospital, London, UK Professor Oliver Gross, University Medicine Göttingen, D Professor Clifford Kashtan, University of Minnesota, USA Professor Jeffrey Miner, Washington University in St. Louis, USA Professor Judith Savige, University of Melbourne, AUS Professor Neil Turner, University of Edinburgh, Royal Infirmary of Edinburgh, UK INTERNATIONAL WORKSHOP ALPORT SYNDROME SEPTEMBER 25 27, 2015 GÖTTINGEN Conference center at the Historic Gauss Observatory includes KDIGO Controversies Conference for advancing towards KDIGO Guidelines Representatives from the following national Alport family support organizations: Dave Blatt, Alport Foundation of Australia Susie Gear and Jules Skelding, Alport support group, UK Sharon Lagas, Alport Syndrome Foundation, USA Daniel Renault, AIRG France/Europe Thomas Simon, Alport Selbsthilfe, D in cooperation with Congress Venue Tagungszentrum an der Sternwarte Geismar Landstr. 11 37083 Göttingen Congress Organization and Registration Aey Congresse GmbH Seumestr. 8 10245 Berlin/Germany phone: 0049 (30) 29006594 fax: 0049 (30) 29006595 e-mail: iwas2015@aey-congresse.de Federation of European Associations of Patients affected by Renal Genetic Diseases supported by the Alport Foundation of Australia and the Associations of Patients from Canada, Spain and Israel More information at www.aey-congresse.de. Page 2 Page 3

Friday, 25 th September 11:00 19:00 hrs Friday, 25 th September 11:00 19:00 hrs Welcome: opening remarks and conference overview 11:00 Welcome and opening remarks Oliver Gross and local committee, Prof. Dr. Heyo Kroemer, Dean of Medical Faculty Göttingen (invited) 11:15 Introduction-round of participants Susie Gear (each participant to shortly introduce her/himself) 12:00 Conference overview getting started Alport syndrome: physichians view Clifford Kashtan Alport syndrome: patients s view Sharon Lagas Road map for the workshop and day 1 Oliver Gross Basic science: Poster session and Panel discussion Chairs: Jeffrey Miner, Dominic Cosgrove, Billy Hudson 14:30 poster-rounds Authors present at their posters (to be presented to audience and chairs) 16:30 Coffee in parallel: European patient associations/federg Meeting expanding cooperation of associations and groups of Europe 17:00 Selected junior speakers with hot topic basic science abstracts Poster Speaker 1 to 4 Basic Science: Plenary Session and Overarching Key-Lectures Chairs: Jeffrey Miner, Billy Hudson 17:45 Round-table discussion of basic science posters Scientific committee and audience 12:40 Extracellular Matrix and cell-matrix interactions Mats Paulsson, head of biochemistry dep, Cologne (invited) 13:00 Matrix biology and cell adhesion Martin Humphries, Dean of Life Sciences, Manchester (invited) 13:30 Lunch 18:45 Summary and completion of goals for basic science breakout group Define bullet points of the basic science group breakout group on day 2 19:00 end of day 1 19:30 Dinner at Theaterkeller, Deutsches Theater Page 4 Page 5

Saturday, 26 th September 09:00 19:00 hrs 09:00 Outline and roadmap for day 2 Oliver Gross and scientific committee Clinical Science: Plenary Session and Overarching Key-Lectures Chairs: Judy Savidge, Frances Flinter, Manfred Weber, Neil Turner Saturday, 26 th September 09:00 19:00 hrs 14:15 Round-table discussion of basic science posters Scientific committee and audience 15:15 If we had unlimited time, brains and money: my questions for you Oliver Gross, Göttingen 09:15 Gene therapy in Cystic Fibrosis: Rationale and clinical trials Eric Alton, Imperial College London (invited) 09:35 Cell based human therapy trials for Duchenne Muscular Dystrophy Guilio Cossu, Manchester (invited) 10:05 Potential therapeutic targets in Alport s 10:30 Coffee syndrome Hans-Joachim Anders, Munich (invited) Clinical science: Poster session and Panel discussion Chairs: full scientific committee 10:45 poster-rounds Authors present at their posters (to be presented to audience and chairs) 12:30 Lunch in parallel: International Patient Associations Meeting future of collaboration and respective implications in research projects and infrastructure development (Registry) 15:45 Coffee Breakout Session I: Group discussion Chairs: Cliff Kashtan, Oliver Gross 16:00 Breakout Group 1: Basic and Translational Science Jeff Miner, Rachel Lennon Breakout Group 2: Diagnosis, Genetics and Gene/Chaperon Therapy Frances Flinter, Constantinos Deltas Breakout Group 3: Standard of care, Repurposing Therapy, Carriers, Management of Extrarenal Symptoms Neil Turner, Christoph Licht, Michelle Rheault Breakout Group 4: Future Therapy, Registries, Biobanks, Clinical Trials Colin Baigent, Tim Friede, Judy Savidge 18:00 Brief summary and completion of goals for breakout groups Discussion lead by C. Kashtan, O. Gross: Bullet points for breakout groups on day 3 13:30 Selected junior speakers with hot topic clinical science abstracts Poster Speaker 1 to 4 19:00 Short dinner at Historic Gauss Observatory 19:30 Guided tour of the historic observatory and guided city tour (mandatory) Page 6 Page 7

Sunday, 27 th September 09:00 13:30 hrs 09:00 Outline and roadmap for day 3 Cliff Kashtan and Oliver Gross Final Breakout Session II: Discussion and Preparation of Recommendations Chairs: Cliff Kashtan, Oliver Gross 09:15 Breakout Group 1: Basic and Translational Science Jeff Miner, Rachel Lennon Breakout Group 2: Diagnosis, Genetics and Gene/Chaperon Therapy Frances Flinter, Constantinos Deltas Breakout Group 3: Standard of care, Repurposing Therapy, Carriers, Management of Extrarenal Symptoms Neil Turner, Michelle Rheault Breakout Group 4: Future Therapy, Registries, Biobanks, Clinical Trials Colin Baigent, Tim Friede, Judy Savidge Sunday, 27 th September 09:00 13:30 hrs 11:30 Breakout Group 3: Standard of care, Repurposing Therapy, Carriers, Management of Extrarenal Symptoms Neil Turner, Michelle Rheault 12:00 Breakout Group 4: Future Therapy, Registries, Biobanks, Clinical Trials Colin Baigent, Tim Friede, Judy Savidge 12:30 Final Prioritization and Conference 13:30 Lunch 14:30 End Summation Cliff Kashtan and Oliver Gross 14:30 Roundtable: Scientific Committee Conclusions and Next Steps 10:15 Coffee Plenary Session: Final Breakout Groups Reports Chairs: Cliff Kashtan, Oliver Gross 10:30 Breakout Group 1: Basic and Translational Science Jeff Miner, Rachel Lennon 11:00 Breakout Group 2: Diagnosis, Genetics and Gene/Chaperon Therapy Frances Flinter, Constantinos Deltas Page 8 Page 9

Invited Speakers Prof. Dr. Heyo Kroemer, Dekan der Medizinischen Fakultät Göttingen, D Prof. Dr. Hans-Joachim Anders, Ludwig-Maximilians-Universität München, Medizinische Klinik und Poliklinik IV, Nephrologisches Zentrum, München, D Prof. Dr. Martin Humphries, Dean of Life Sciences, University of Manchester, UK Prof. Dr. Leena Bruckner-Tuderman, Ärztliche Direktorin der Universitäts-Hautklinik Freiburg, Vizepräsidentin der DFG Prof. Dr. Eric Alton, Imperial College London, Professor of Gene Therapy and Respiratory Medicine, UK Prof. Dr. Giulio Cossu, Manchester; Professor of Human Stem Cell Biology at University College London, UK Prof. Dr. Matthias Kretzler, University of Michigan, Medical School, Internal Medicine, Nephrology, Michigan, USA Page 10 Page 11

Arrival Keplerstraße Rathaus/ Town Hall Historische Sternwarte/ Historic Observatory Vom Bahnhof kommen Sie mit dem Bus Nr. 12 (Richtung Geismar-Schöneberger Str. ) zur Haltestelle Keplerstraße. Zu Fuß erreichen Sie die Sternwarte vom Bahnhof aus durch die Innenstadt in etwa 25 Minuten. Sie gelangen dann sowohl über die Keplerstraße als auch über die Geismar Landstraße 11 (hier der kleine Weg schräg gegenüber dem Neuen Rathaus) in den der Stadt zugewandten Innenhof der Sternwarte und zum Lichtenberg-Kolleg. Im Innenhof gibt es leider keine Parkmöglichkeiten für PKW. From the train station you can either take the Bus Nr. 12 (Direction: Geismar-Schöneberger Str. ) to Keplerstraße or walk to the Observatory in ca. 25 minutes via the city centre. Via Keplerstraße or Geismar Landstraße 11 (take the alleyway opposite the New Town Hall) you reach the inner court yard and the Lichtenberg-Kolleg. Please note: There are no car parking spaces in the inner court yard. Page 12 Page 13

Arrival Airport: Frankfurt There are direct and fast train connections (Frankfurt-Airport - Göttingen by ICE: approx. 2,5 hours). From the train station you can either take the Bus Nr. 12 (Direction: Geismar-Schöneberger Str. ) to Keplerstraße or walk to the Observatory in ca. 25 minutes via the city centre. Via Keplerstraße or Geismar Landstraße 11 (take the alleyway opposite the New Town Hall) you reach the inner court yard. Please follow the road out of town, called Geismar Landstraße, choose the first entrance on the left side (in front of the large wall). From the first entrance you have to go ahead. If you have reached the Courtyard please go straight ahead and right at the end of the way. Within in the yellow building you will find some of the course rooms. Please note: There are no car parking spaces in the inner court yard. Accomodation Eden Hotel Reinhäuser Landstraße 22a 37083 Göttingen Single room incl. Breakfast 104 For your hotel reservation please contact Mrs. Grit Lommatzsch via e-mail g.lommatzsch@aey-congresse.de Page 14 Page 15