7th Rare Disease Summer School

from the University of Geneva with the poster: "Targeting RNA Conformational Ensemble for Developing Therapeutics against Rare Disease: The Hutchinson-Gilford Progeria Syndrome"

from the University Hospital Zurich with the poster: "Investigating the intracellular communication networks in the epidermis that drive pathogenesis of the skin disorder Pityriasis rubra pilaris"

"I liked the multidisciplinary environment and the possibility to network with people coming from different fields. I learned what - bed to bench, bench to bed - means and that collaboration is crucial for researchers, affected families, and regulators. This important information was not part of my knowledge before."

"I enjoyed very much the intensive poster discussion on the first day and the involvement of patients in presenting and showing their engagement in research and drug development. This in a very open-mind setting and good learning environment."

Dr. Jasmin Barman-Aksözen, Scientific advisor, Swiss society for porphyria: Clinical trial design and approval process
Prof. André Brändli, Ludwig-Maximilians-Universität München:
Drug discovery
Dr. Guillaume Canaud, Necker Hospital, Paris, France: Targeted therapy in patients with PIK3CA related overgrowth syndrome
Prof. Andrew Dwyer, Boston College, Boston, USA:  Reproductive endocrinology 
Christina Fasser, Vice-President ProRaris, President Retina International: Patient-initiated research
Prof. Nine Knoers, Chair Dept. of Genetics, University Medical Center Groningen, the Netherlands 
Prof. Stephan Neuhauss, University of Zurich: Model organisms
Dr. Jürg Streuli, Children's Hospital Zurich: Ethical considerations
Prof. Marshall Summar, Children‘s National Medical Center, George Washington University, Washington DC, USA: Clinical endpoints
Dr. Martine Zimmermann, Alexion Pharma International: Regulatory frameworks