The conference sessions will explore issues and cutting-edge technologies that affect many adult and pediatric conditions. The following topics are to be discussed: • Drug repositioning and personalized medicine • NGS and undiagnosed rare diseases • Pathophysiology • Bringing treatments to the clinic • Neurological diseases • Patients and research Our aim is to promote research on rare and orphan diseases among the general public, industry and policymakers as well as to provide a forum for researchers to meet and pool their knowledge. The underlying objective is to tackle the key issues that need to be addressed if we are to see new and promising therapies and treatments rapidly delivered to patients all around the world. This will also help to further understanding of other more common diseases and to encourage clear insights from the scientific community in universities and industry. The congress will bring together world leaders and young scientists from stem cell, cell biology, gene therapy, human genetic, or therapeutic applications to present state-of-the-art research, to discuss results and to exchange ideas.
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