Presentations and Workgroup Reports

Inaugural of Conference
Kerstin Westermark (Sweden, COMP, EU) and Stephen C. Groft (Office of Rare Diseases Research, NIH, USA)
Actions on Rare Diseases from a Pioneer Country Toward the Rest of the World. A Vision of Globalization.

Initiatives from the Public Institutions to Research and Patients Access
Erik Tambuyzer (Genzyme Corporation, Belgium)
History and Commitment from industry in the field of rare diseases and orphan drugs.

Research on Rare Diseases at Academia and in Industry
Jan-Inge Henter, Annika Larsson and Désirée Gavhed (Karolinska Institute/Karolinska University Hospital, Sweden)
Added Values of Research on Rare Diseases.
Erik Tambuyzer (Genzyme Corporation, Belgium)
Business models

Bioethics in vulnerable populations
Luis Alejandro Barrera (University Javeriana, Colombia)
Ethics and vulnerable population

Linking needs from neglected diseases
Sharon Terry (Genetic Alliance, USA)
Gateway to Rare and Neglected Disorders Therapeutics
Alberto Mantovani (Italian National Institute of Health, Italy)
Food Components and Rare Birth Defects

Exploring Strategies for a Better Accessibility to Diagnosis and Therapies
Sonia van Weely (Dutch Steering Committee on Orphan Drugs, Netherlands)
Orphan drug regulation and national strategies: complementary actions
Carlos Correa (University of Buenos Aires, WHO, Argentina)
Intellectual Property and Needs for the Development of Neglected Medications.

International Initiatives on Rare Diseases and Orphan Drugs
Emilio J. A. Roldán (SLADIMER)
International initiatives on rare diseases and orphan drugs ARGENTINA
Verónica García and Francesc Palau (CIBERER)
Biomedical Network Research Centre on Rare Diseases, A model of cooperative research on Rare Diseases

Plenary: Development of information in Rare Diseases and orphan products
Jan-Inge Henter (Karolinska Institutet, Sweden)
Role of Academia in Rare Diseases

Working group presentations and reports

Working group on Research Collaborations
Coordinators: Emilio Roldán (Argentina) and Karina Brito (Latin America)
Barbara Wuebbels (Biomarin, USA)
Orphan drug development

Working group on Obtaining the Diagnosis of Rare Diseases
Coordinators: Domenica Taruscio (Italy) and Manfred Stuhrmann (Germany)
WG Report Obtaining the Diagnosis of Rare Diseases

Working group on Food, Environment and Rare Diseases
Coordinators: Alberto Mantovani (Italy) and Manuel Posada (Spain)
WG report Food and Environment

Working group on Patient/Family Needs, Disease Awareness, and Informational Needs
Coordinators: Sharon Terry (USA) and Cecilia Micheletti (Brasil)

Working group on Access to Therapy in Countries With or Without Orphan Drug Legislation
Coordinators: Erik Tambuyzer (Belgium) and Alejandra Menendez (Argentina)

Working group on History, Philosophy and Ethics
Coordinators: Bernardino Fantini (Italy), Segolène Ayme (France) and Luis Alejandro Barrera Avellaneda (Colombia)
WG Report List of the Top Ten Ethical Tensions and Philosophical Issues

Working group on Regulatory Needs
Coordinators: Timothy Coté (USA) and Kerstin Westermark (Sweden)

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