Novartis Pharma / Merck KgaA / Sanofi Company / AKU Society / EU Genzyme / Vice-Chair COMP / Alexion Pharmaceuticals / Agency for Medicinal / Genzyme / Pfizer / Haffner Associates / Next Generation Sequencing Diagnostics / European Reference Networks / Building European Reference Networks / Orphanet Journal / Molecular Genetic Diagnostics / DIA Europe / Eastern European Health Care Systems / UK Patients / Actelion / Health / Vertex / Emeritus / Alström Syndrome / Orphanet-Inserm / HAS’ / Medical / /
Continent
Africa / Europe / /
Country
United Kingdom / Italy / Spain / Netherlands / France / Canada / Germany / Romania / Sweden / Belgium / Austria / United States / Czech Republic / /
Event
Business Partnership / FDA Phase / /
Facility
Rare Diseases Coordinating Centre / Leiden University Medical Center / Radboud University Nijmegen Medical Centre / National Institute / Genomics Education Centre / Johann Wolfgang Goethe University / University Medicine Berlin / Necker Hospital / Observatory Study / University Medical Center / UK European Bioinformatics Institute / European Bioinformatics Institute / Institute of Genetic Medicine / Medical University of Vienna / Charles University / Federal Institute / German Institute of Medical Documentation / University Gasthuisberg / Institute of Genetic Medicine Newcastle University / Scientific Institute of Public Health / University of Mainz / University of Cambridge / University of Tübingen / Clinical Institute of Neurology / University Salzburg / Belgium National Institute of Health / Leiden University Medical Centre / Albert-Ludwigs University of Freiburg / Children’s Hospital of Eastern Ontario / Paris Descartes University / University Hospitals Leuven / Academic Medical Center / Academic Medical Centre / National Health Care Institute / Aarhus University Hospital / Wellcome Trust Sanger Institute / Newcastle Upon Tyne Hospital / Technical University of Denmark / European Observatory / Institute of Rare Diseases / /
IndustryTerm
Rare Disease Therapy / development chain / broader healthcare system / specialised management / healthcare pathways / regulatory tools / health information systems / therapeutic medicinal products / healthcare system / mainstream media coverage / healthcare outcomes / RARE DISEASE Therapies / healthcare delivery / therapy for rare disease / medicinal products / operational diagnostic and therapeutic tools / 200 New Rare Disease Therapies / treatment for rare diseases / broader network / Web Communications Senior / crossborder healthcare / medical devices / genetic alliances network / healthcare / healthcare systems / regulatory and other tools / biotech/pharmaceutical partners / orphan medicinal products / /
MedicalCondition
Alström Syndrome / Most Rare Diseases / needed rare disease / diseases / Inherited Metabolic Diseases / Parent Project Muscular Dystrophy / disease / Association Française contre Les Myopathies / Rare Diseases / Austrian Epidermolysis Bullosa / Developmental Disorders / Hemiplegia / epidermolysis bullosa / Cystic Fibrosis / single disease / rare disease / /
MedicalTreatment
stem cell therapy / /
Organization
Internal and External Communications Unit / Technical University of Denmark / Newcastle Upon Tyne Hospital / Wellcome Trust Sanger Institute / World Health Organization / UK European Bioinformatics Institute / Centre for Medical Systems Biology / Scientific Institute of Public Health / Department of Dermatology / APHP / EHC Steering Committee / Children’s Hospital of Eastern Ontario / Joint Research Centre / Neuromuscular GeneticsMRC Centre for Neuromuscular Diseases / Alliance for Chronic Rare Diseases / Romania Prader Willi Association / Germany Public / Italian Medicines Agency / Centre of Rare Diseases / FDA / Necker Hospital for Children / Academic Medical Center / Dutch Federation of Cancer Patient Organisations / Hopital Necker / National Health Service / French Centre / Department of Clinical Pharmacology / Directorate for Health Research / Committee for Orphan Medicinal Products / Clinical Institute of Neurology / Programmes and Stakeholders Relations Directorate / European Genetic Alliance / National Health Care Institute / Academic Medical Centre / Leiden University / Genetic & Rare Disorders Organisation / German Institute of Medical Documentation and Information / Core Unit for Applied Genomics / University Hospital / Medical Products Agency / DLR Project Management Agency / Society for Mucopolysaccharide Diseases / University of Tübingen / Albert-Ludwigs University of Freiburg / Health Research Directorate / International Centre for Life / National Institute for Health and Welfare / London School of Economics / Johann Wolfgang Goethe University / University of Mainz / Medicines and Healthcare Products Regulatory Agency / Aarhus University Hospital / Federal Agency for Medicinal and Health Products / Business Contact Centre / Oncology Unit / Paris Descartes University / French National Authority for Health / Genomics Unit / Committee of Orphan Medicinal Products / Committee on Reimbursement of Drugs / Institute of Rare Diseases / European Commission / Medicine Foundation / European Gaucher Alliance / European Union / Institute of Genetic Medicine / Institute of Genetic Medicine Newcastle University / NHS / Belgium National Institute of Health / Ministry of Health / Rare Diseases Coordinating Centre / Laboratory for Molecular Diagnosis / Rare Disease Foundation / Faculty of Medicine / University Medical Center / Federal Institute for Drugs and Medical Devices / European Patients’ Academy on Therapeutic Innovation / National Centre for Rare Diseases / Italy Emergency Guidelines and Emergency / Product Development Scientific Support Department / Austrian Social Securities Association / Terol / Policy Officer / DG SANCO / Health and Consumers Unit / Leiden University Medical Centre / Berlin Center for Rare Diseases / Radboud University Nijmegen Medical Centre / Medical Classifications Unit / Consumers Unit / Scientific Advice Working Party / Organisation of Healthcare Pathways / National Genetics and Genomics Education Centre / Executive Office / Charles University / European Medicines Agency / France Off-label Use / MRC Centre for Neuromuscular Diseases / University of Cambridge / Chair Genetic & Rare Disorders Organisation / Romanian National Alliance / University of Vienna / Centre of Expertise / Center for Human Genetics / Spanish Federation of Rare Diseases / office of Orphan Products Development / /
Person
Denis Costello / Spiros Vamvakas / Thomas Wagner / André Lhoir / Centre / Luca Pani / Gert Matthijs / Rémy Choquet / Véronique Héon-Klin / Edmund Jessop / Milan Macek / DG PRE / Tony Hall / Enrique Terol / Innovative Medicines / Epidemiology / John Wilkinson / Kris De Boeck / Therapies Jordi Llinares Garcia / John F Ryan / Wills Hughes-Wilson / Jade Improving / Findacure / Emmanuelle Lecomte-Brisset / Variation / Patients Phil Beales / Jeremy Manuel / Nick Sireau / Kym Boycott / Christine Lavery / Arnaud de Villeneuve / Marlene Haffner / Durhane Wong Rieger / Peter Bauer / Leena Bruckner-Tuderman / Flaminia Macchia / Odile Kremp / Fabry Shortage Carla Hollak / Damian Smedley / Gauthier Chassang / Domenica Taruscio / Lesley Greene / Jytte Lyngvig / Christoph Nachtigäller / Jade Understanding / Thomas Heynisch / Erik Tambuyzer / Hélène Dollfus / Debra Lewis / François Houyez / Patients Maria Mavris / Yann Le Cam / Pierre Sarda / Bruno Sepodes / Nathalie Seigneuret / Ana Rath / Avril Daly / Orpha Codes / Orphanet / Jordi Llinares Garcia / Ri de Ridder / Fighting Blindness / Opal Pre-competitive / Elizabeth Vroom / Rare Kym Boycott / Heidi Wagner / Genetic / Carla Hollak / Fondazione / Enfants Malades / Breakthroughs Christine Mummery / It Dorica Dan / Ciaran Nicholl / Daniel Rosenberg / Patricia Hurter / Peter Rutherford / Ad Schuurman / Carlo Incerti / Frank Ückert / Kerstin Westermark / Kate Bushby / Hermann Gröhe / Ole Winther / Stefan Schreck / Adam Heathfield / Alström / Gabi Pohla-Gubo / Christine Mayer-Nicolai / Marc Dooms / Pauline Evers / Hanns Lochmüller / Sabine Sarnacki / Daria Julkowska / Brigitte Bloechl-Daum / Ségolène Aymé / Baxter Healthcare / Paolo Siviero / Manuel Posada / Serge Braun / Peter Farndon / Maria Pascual / François Meyer / Irene Norstedt / Yves Juillet / DG SANCO / Kay Parkinson / Peter Robinson / Pat Furlong / Innovative Medicines Initiative / /
Position
Federal Minister / Professor of Inherited Metabolic Diseases / Advisor / Associate Professor / DTU Informatics / Chief Executive / Vice President / Head / Head of Scientific Advice / Physician / Acting Head / Editor in Chief / General / Director General / Associate Professor and Deputy Head / Chief Scientific Officer / Benefit-Risk Assessment Session Chair / Medical Specialist / Senior Scientific Project Manager / Head of Unit / Professor / Director Public Health / DG Health & Consumers / Dissemination Development Officer / Managing Editor / Director - Europe / FAVOURABLE ECO-SYSTEM Session Chair / Director / EURORDIS / France Panel Discussion / Vice-President / EURORDIS / Director International Affairs / Investigator / Deputy Head / Team Leader / Director Medical Affairs / Director / Worldwide Policy / Medical Director / Minister of Health / Director of Research / Director of International Affairs / Vice President Medical and Clinical Operations & Regulatory Affairs / Professor of Developmental Biology / Senior Vice President / Global Pharmaceutical Development / unmet medical needs Session Chair of product development TO ADDRESS / model / Manager / Conectus / France Session / Senior Scientific Advisor / Professor of Medical Genomics / Lawyer / Project Leader / Chair / UK Session / RU Session Chair / Manager / OrphanAnesthesia / Germany Harmonisation / President / Director / Global Program Regulatory / Professor and Chair / Project and Investment Manager / Chief Executive Officer / Manager / National Bank / Drug Session Chair / Diagnostic Services Session Chair / Chair / UK BREAKTHROUGHS IN SCIENCE Session / Chair / International Rare Diseases Research Consortium / products Session Chair / challenges Session Chair / Head / Advocate / Access Officer & Vice President External Affairs / Vice President Global Public Policy & Government Relations / Senior Orphan Drug Specialist / Policy Officer / Directorate-General / Research Officer / Secretary General / Academy / Head of Economic Strategy and Pharmaceutical Policy / Access Director / treating physician / Chief Commercial Officer / diseases Session Chair / Associate Professor / General Practitioner / Director / EURORDIS / France Session / Director of Devices / Ispra Sector Head / Policy Officer / Information and Access Director / Co-Founder / Project Manager / Vice-President / CLIMB / representative & Chief Executive Officer / Scientific Administrator / Session Chair / moving forward / President / International Affairs / Head of Economic Strategy & Pharmaceutical Policy / Directorate-General for Health and Consumers / Professor of Medical Genetics / Chairman / Coordinator / Deputy Director / Director / Head / Director / Director Therapeutic Development / Director BioBanking and Biomolecular Research Infrastructure / Manager & RareConnect Leader / Chair / Head International Regulatory Strategy / Senior Vice President / Global Government Affairs / adviser / Vice-President / EURORDIS / Chair / Professor of Neuromuscular Genetics / Chair of Experimental Myology / Head of Medical Informatics / Coordinator of EuroGentest / Chief / Coordinator of expert centre on / Member / Director European Public Affairs / Vice-Chair / Vice-President / CLIMB / Committee / forward / /
Product
Marc Dooms / International / /
ProvinceOrState
Ontario / Veneto / /
PublishedMedium
Chief / Orphanet Journal / /
Region
Eastern Ontario / Middle East / /
Technology
Biotechnology / cell therapy / 3 EMA-HEALTH TECHNOLOGY / Stem Cells / Genomics / Bioinformatics / drug development / /
OPENING SESSION Friday, 9 May[removed] – 09.45 I Rubin Room (Level -1) The Opening Session will be simultaneously interpreted from English into 5 languages: