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THIS SITE IS INTENDED FOR HEALTHCARE PROFESSIONALS ONLY.
Sponsorship statement: This is a non-promotional meeting funded and organised by Sarepta. These presentations and discussions are for educational purposes only and solely intended to provide scientific information on certain diseases. Nothing in these presentation and discussions should be construed as seeking to promote any of Sarepta’s therapies. Nothing in these presentations should be disclosed externally without Sarepta’s prior agreement.
Live Webinar
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Pan-European Series
The importance of gene therapy in major therapy areas is increasing year on year, and consequently gene therapy trials are being instigated. Yet relatively few centres across Europe have experience of running clinical trials in this complex area.
This series of webcasts will provide a forum for sharing best practice on the core aspects of running AAV gene therapy trials. The series begins with a webcast on the background to gene therapy trials, the differences between these and traditional clinical trials, and the key advice you need to establish a new trial site. The webcast will include presentations by experts from across Europe, as well as panel discussions and the opportunity for you to pose your questions to the experts in live Q&A sessions.
Practical experiences from running an adenoassociated virus gene therapy trials: The Principal Investigator’s perspective
XX September 2020 | 18:00 GMT/19:00 CET
Professor of Paediatric Neurology, Director of the Dubowitz Neuromuscular Centre, at the UCL Institute of Child Health and Great Ormond Street Hospital for Children, London, UK
Professor of Paediatric Neurology at the Catholic University, Rome, Italy
Professor of Paediatric Neuromuscular Diseases at the MDUK Oxford Neuromuscular Centre, Invited Professor of Child Neurology at Liège University.
Chef de clinique neurologie pédiatrique Hôpital Universitaire Des Enfants Reine Fabiola (HUDERF), Brussel, Belgium
FULL BIO +
Head of the centre of pediatric neuromuscular diseases at the v. Hauner children hospital, University of Munich, Munich, Bavaria, Germany
FULL BIO +
Professor of Paediatric Neurology, Director of the Dubowitz Neuromuscular Centre, at the UCL Institute of Child Health and Great Ormond Street Hospital for Children, London, UK
Professor of Paediatric Neurology at the Catholic University, Rome, Italy
Professor of Paediatric Neuromuscular Diseases at the MDUK Oxford Neuromuscular Centre, Invited Professor of Child Neurology at Liège University.
Professor of Paediatric Neurology, Director of the Dubowitz Neuromuscular Centre, at the UCL Institute of Child Health and Great Ormond Street Hospital for Children, London, UK
Professor of Paediatric Neurology at the Catholic University, Rome, Italy
25 minutes
Welcome, intro to the meeting and agenda, instructions for delegates
A brief history of gene therapy trials in Europe
Gene therapy trials vs traditional trials – what are the differences?
Reflections on successful gene therapy trials
Practical guidance on running multiple gene therapy trials at a single site
Speaker(s):
Prof Francesco Muntoni
Prof Eugenio Mercuri
15 minutes
Speaker(s):
Prof Laurent Servais
20 minutes
Personal experiences of trial management – what worked and what didn’t?
Speaker(s):
Prof Francesco Muntoni / Prof Eugenio Mercuri (chairs)
Prof Laurent Servais / Prof Nicolas Deconick /
Prof Wolfgang Müller-Felber (faculty)
30 minutes
Chairs:
Prof Francesco Muntoni
Prof Eugenio Mercuri
Sponsorship statement:
This is a non-promotional meeting funded and organised by Sarepta. These presentations and discussions are for educational purposes only and solely intended to provide scientific information on certain diseases. Nothing in these presentation and discussions should be construed as seeking to promote any of Sarepta’s therapies. Nothing in these presentations should be disclosed externally without Sarepta’s prior agreement.
Job code: XXX | Date of preparation: August 2020
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COPYING OF THIS MATERIAL BY ANY MEANS WITHOUT SAREPTA’S PRIOR WRITTEN CONSENT IS PROHIBITED
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