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17 Mar 2014

Orphan Diseases and Back Translation Approach for Drug Discovery and Development

Minisymposium arranged by Drug Research Academy, UCPH and Orphan Disease Council

To be held in Copenhagen on Thursday 20th of March 2014 at Benzon Auditorium, PharmaSchool,Universitetsparken 2, Copenhagen Ø

Faculty of Health and Medical Sciences, UCPH

The meeting is open for interested stakeholders. No registration is necessary.

Orphan Diseases and Back Translation Approach for Drug
Discovery and Development

13.00 – 13.05 Welcome. Ole J. Bjerrum, PharmaSchool, UCPH

13.05 – 13.15 Orphan Drugs and Orphan Disease Council Lisbeth Thyregod, Chair Orphan Disease Council

13.15 – 13.45 Rationales for development of new orphan drugs. Martin Bonde, CEO, Epitherapeutics, Copenhagen

13.45 – 14.25 Post-transcriptional control processes – a new technology in development of drugs for orphan diseases. Exemplified by Duchennes muscular dystrophy Stuart Peltz, PTC Therapeutics, USA

14.25 – 15.00 On the development of a drug for treatment of Niemann-Pick’s disease, type C. Thomas Kirkegaard, CSO, Orphazyme, Copenhagen

15.00 – 15.20 Coffee

15.20 – 15.55 Phase 3 intervention study with Lamazyme on patients with alfa-mannosidosis Christine Dali, Dept. of Clinical Genetics, Rigshospitalet, Copenhagen

15.55 – 16.30 Use of patients with Dopa responsive dystonia to establish proof of concept for new target in pain treatment. Arafat Nasser, PharmaSchool, UCPH

16.30 – 16.45 Conclusion and Perspective, Ole J. Bjerrum, PharmaSchool, UCPH

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