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Pauline Samia
Article
Position Statement:Emerging genetic therapies for rare disorders
Journal of the International Child Neurology Association
  • Pauline Samia, Aga Khan University
  • Adam Kirton, University of Calgary
  • Russell Dale, The University of Sydney
  • Silvia Tenembaum, National Paediatric Hospital
  • Chahnez Charfi Triki, Université de Sfax
  • Anaita Hegde, Jaslok Hospital and Research Centre, Mumbai
  • Richard Idro, Makerere University College of Health Sciences
  • Jo M. Wilmshurst, University of Cape Town, South Africa
  • Ingrid Tein, University of Toronto, Canada
  • Haluk Topaloğlu, Hacettepe University School of Medicine in Ankara
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Publication Date
7-1-2019
Document Type
Article
Disciplines
Abstract

Emerging genetic therapies for rare disorders at high cost, cannot realistically address the global burden of disease. Stakeholders must develop new pathways to ensure safe, fair and sustainable provision of such therapies.

Creative Commons License
Creative Commons Attribution 4.0 International
Citation Information
Pauline Samia, Adam Kirton, Russell Dale, Silvia Tenembaum, et al.. "Position Statement:Emerging genetic therapies for rare disorders" Journal of the International Child Neurology Association Vol. 1 Iss. 1 (2019)
Available at: http://works.bepress.com/pauline_samia/13/