List of confirmed speakers
Ronald Frank, Leibniz Institute for Molecular Pharmacology, Berlin (Germany): EU-OPENSCREEN Research Infrastructure
Bill Gahl, Undiagnosed Diseases Program, NIH, Washington (USA): Clinical diagnosis of rare diseases
Richard Gregory, Head of Genzyme R&D Center (USA): Innovation in Rare Disease R&D
Michael Hayden, President of Global R&D and Chief Scientific Officer, Teva Pharmaceutical Industries (Israel): From rare diseases to treatment of common disorder
Dan Kastner, Director of Intramural Research, NIH, Washington (USA): Horror Autoinflammaticus: The Expanding Spectrum of Systemic Autoinflammatory Diseases
Petra Kaufmann, Director of the Division of Clinical Innovation at NIH`s National Center for Advancing Translational Sciences (NCATS), NIH, Washington (USA): NCATS – Catalizing Translational Innovation
Jürgen Knoblich, Institute of Molecular Biotechnology, Vienna (Austria): Using 3D culture organoid models to model rare brain disorders
Hanns Lochmüller, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne (UK): RD-Connect
James Lupski, Baylor College of Medicine, Houston (USA): Genome-wide diagnosis of rare diseases
Shoukhrat Mitalipov, Oregon Health & Science University, Portland (USA): Germline gene therapy of mitochondrial diseases
Josef Penninger, Institute of Molecular Biotechnology, Vienna (Austria): Model system for diagnosis and therapies of rare diseases
Ole B. Suhr, Umea University, Umea (Sweden): Development of treatment of transthyretin amyloidosis, safety and efficacy of Si-RNA treatment
Takanori Takebe, Department of Regenerative Medicine, Yokohama University, Yokohama (Japan): Pluripotent stem cells and organ reconstitution for rare diseases
Selected Presentations from the Research Networks on Rare Diseases in Germany.
With involvement of patient organisations and pharmaceutical industry.