David Dasberg is the Managing Director of Facio Therapies and Facio Intellectual Property. He has over 20 years' experience with the development and commercialization of (bio)pharmaceutical products. As an independent consultant, David performed a feasibility study commissioned by the Dutch...

Wills Hughes-Wilson is Chief Patient Access Officer at Sobi and a member of the company’s global Executive Management team.  She joined the company in 2012. Hughes-Wilson leads the cross-functional, international team responsible for Sobi’s patient-access-centred approach across the company and its ...

Following a Ph.D. in Biochemistry and Molecular Biology from the University of Zürich Switzerland, Dr Linden held faculty positions at Mount Sinai School of Medicine in New York and King’s College London, engaged in the field of AAV biology and AAV-mediated gene therapy.  Most recently Michael...

Mapi is the only Global CRO specializing in late phase research. We also provide strategic consulting and related services in Epidemiology and Product-Related Risk Management . This includes market forecasting, health burden and economic assessments, pharmacovigilance and risk management plans, and ...

Diana has played a leading role at Action Duchenne for the past six years and was appointed as CEO last year, based on her knowledge and invaluable contribution to the Duchenne community. She has worked closely with hundreds of international families and young people living with Duchenne and other...

Paul is Executive Director in the Office of the Chief Patient Officer at MSD, a global healthcare company with a 125-year history of working to make a difference to people’s lives.  He has held posts in clinical research and medical affairs in both the UK and the USA.  He has a background in...

Sheela Upadhyaya is currently the Associate Director of the Highly Specialised Technology program at NICE and is responsible for running the program to evaluate medicines and technologies for rare and ultra-rare conditions for commissioning in the NHS.Prior to joining NICE, she was responsible for...

Dr. Stephen Yoo is the Chief Medical Officer of REGENXBIO. Prior to joining REGENXBIO, he was Medical Science Director of Clinical Development at AstraZeneca and Group Director of Clinical Development at MedImmune, AstraZeneca’s global biologics research and development arm. In these roles, he led...

Omar Khwaja has been Global Head of Rare Diseases at Roche since 2015. He joined Roche in 2011 as a Translational Medicine Leader responsible for the early development of new medicines for neurodevelopmental disorders and rare diseases,. His group is responsible for the discovery and development to ...

Bruno Sepodes holds a PhD in Pharmacy (Pharmacology) by the University of Lisbon. Currently a Professor of Pharmacology and Pharmacotherapy at the Faculty of Pharmacy of the University of Lisbon, he develops his research in Pharmacology and Translational Medicine. Presently Prof. Sepodes is the...

Julie Walters, founder, is the driving force behind Raremark and leads on product strategy. She has a first-class degree in molecular genetics from King’s College London and a background in popular journalism, including time as a news editor on TV’s Good Morning Britain. Outside Raremark, Julie is ...

Dr. Christina Waters has been responsible for leading medical research in a broad range of organizations ranging from academia and disease – focused non-profits to biotech and large pharmaceutical companies.   Her broad range of experience in basic, translational and clinical research and business...