Conference date: Monday 10th July 2017
If you are a doctor, scientist or patient representative you may request an invitation by emailing firstname.lastname@example.org
We are planning to make this conference as successful as possible and we expect to help shape MSD research for the near and foreseeable future. This conference will help to foster collaboration among expert scientists and doctors.
Prof. Dr. Andrea Ballabio Director of TIGEM, Naples, Italy. Co- founder of the SUMF1 gene that is deficient in MSD.
Speaker: SUMF1: The master and commander of sulfatases.
Prof. Dr. Kurt Von Figura Children’s University Hospital Gottingen, Georg-August-Universität Gottingen, Germany. Co-founder of the SUMF1 gene that is deficient in MSD
Dr. Lars Schlotawa, MD, Postdoctoral Research Associate, University of Cambridge, UK. (Co-Chair of conference)
Speaker: Clinical Aspects of Multiple Sulfatase Deficiency: What we know and what we do not know yet.
Prof. Dr. Gregory Pastores (meeting Chair) Mater Hospital & Temple Street Children’s Hospital, Dublin, Metabolic consultant. (Co-Chair of conference)
Prof. Dr. Thomas Dierks, Head of Facility of Biochemistry, University Bielefeld, Germany
Dr. Karthikeyan Radhakrishnan, Facility of Biochemistry, University Bielefeld, Germany.
Speaker: Recognition and ER quality control of misfolded FGE by PDI: Implications for MSD
Prof. Dr. Jutta Gärtner, Head of Children’s University Hospital Gottingen, Georg-August-Universität Gottingen, Germany
Prof. Nicola Brunetti, Associate Investigator of Molecular Therapy,TIGEM, Naples, Italy.
Speaker: Gene therapy clinical trial for mucopolysaccharidosis type VI
Prof. Giancarlo Parenti, Associate Investigator, TIGEM & Associate Professor of Pediatrics & Department of Translational Medicine, University of Naples “Federico II”, Italy
Dr. Rachel Bailey, Postdoctoral Research Associate, Gene Therapy Center, Gray Lab, University of North Carolina, USA. Speaker: Widespread nervous system gene transfer using AAV9 vectors: our experience with a first-in-human gene therapy trial for Giant Axonal Neuropathy
Dr. Rebecca Ahrens-Nicklas, M.D.,Ph.D. paediatrician at CHOP, Philadelphia, USA
Speaker: MSD iPSC project, introduction & discussion.
Sean Ekins M.Sc., Ph.D. Collaborations Pharmaceuticals & Phoenix Nest, USA.
Speaker: Building companies for rare & neglected diseases with NIH funding /SBIR grant for drosophila screen on SUMF1 mutants
Dr. Florian Eichlar S., MD Professor of Neurology, Head of Leukodystrophy, Massachtusetts General Hospital, Boston, USA. Speaker: Gene therapy approaches in the leukodystrophies
Dr. Laura Adang, MD, Ph.D. Assisting Professor of Child Neurology at CHOP, Philadelphia, USA (specialising in leukodystrophies) Speaker:Comprehensive care of children with leukodystrophies
Angeleen Fleming Ph.D, Senior research Associate Department of Medical Genetics, Cambridge Institute for Medical Research.
Speaker: Using zebrafish models of neurological disorders to identify novel therapeutic pathways.
Dr Matthias Baud, Lecturer in Medicinal Chemistry and Chemical Biology at the University of Southampton.
Matthew Campbell PhD, Research Assistant Professor, Lecturer in Genetics, Smurfit Institute of Genetics,Trinity College, Dublin, Ireland.
Speaker: RNAi mediated modulation of BBB; Enhancing delivery of biological therapeutics.
Dr. David J Begley Kings College London, Institute of Pharmaceutical Science. Vice-President and Scientific Director Brains for Brain Foundation.
Speaker: Strategies for delivering enzyme therapy to the brain.