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Orphan Drugs & Rare Diseases Conference, 8-9 October 2012, London, UK

SMi is delighted to welcome: Carlos , Vice President & Chief Medical Officer at uniQure, to their inaugural & on 8th & 9th October 2012 in . Dr. will be joining leading leaders in and . As recently reported in Nature, UniQure have won recommendation to market the western world’s first , Glybera.  The company’s success is a huge step towards a long-cherished goal of : delivering, in a single treatment, a life-long, functional copy of a missing gene to patients. 

Jorn Aldag, chief executive of UniQure, says that the announcement from the EMA is an “overdue signal” to the gene-therapy community that things are changing. “It unlocks the potential,” he says, quoted in Nature. “You will see more investment coming.”*

Perhaps as important is UniQure’s pioneering “annuity” approach to reimbursement, staggering payments over the five years for which treatment has been proved to work. As highlighted by the Financial Times, such financial innovation may prove as important as the scientific breakthroughs that led to the treatment in the first place**.

Other keynote speakers presenting at this outstanding event include:


  • Geraldine Honnet, Head of Clinical Development, Généthon
  • Celine Plisson, Associate Medical Director, Orphan Europe Recordati Group
  • Gary Clements, Senior Director, Business Development, Shire 
  • Richard Philipson, Disease Area Head for Immuno / Inflammation, GlaxoSmithKline, Neurosciences MDC
  • Elisa Muscianisi, Medical Advisor, Bone, Inflammation & Rheumatology Areas, Novartis


Visit http://www.smi-online.co.uk/goto/orphandrugs-event34.asp to secure your place now! Alternatively, contact Alternatively Contact Fateja Begum on telephone +44 (0) 20 7827 6184 or email on [email protected]

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* Europe nears first approval for gene therapy. Nature News & Comment, July 20th, 2012.


** Healthcare: Uncommon complaints. Financial Times, July 25th, 2012, p.9.