Third IRDiRC Conference on February 8-9, 2017 in Paris, France

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The 3rd International Rare Diseases Research Consortium (IRDiRC) Conference took place in Paris, France, on February 8-9, 2017. Around 300 participants from around the globe, including academics, researchers, industry leaders, policy makers and patient advocates, attended the meeting to help shape the future of rare diseases research.

The conference provided a unique opportunity for stakeholders active in the field of rare diseases research to reflect on the progress made in the last decade, particularly since the launch of IRDiRC in 2011, and to look forward to the challenges ahead. For more details, the conference report will soon be available here.

You can also access the conference book with abstracts.

PowerPoint presentations of the speakers, organized by sessions, are accessible below.

 

Plenary Session 1: Opening Session – IRDiRC History and Achievements

Plenary Session 2: Rare Diseases Research in 2017 – A Global View

Plenary Session 3: State of Foundational, Diagnostics, Therapeutics Research

Parallel Session 1: Diagnostics, Foundational and Therapeutics Research in 2017

Track 1 – Diagnostics Research in 2017

Track 2 – Foundational Research in 2017

Track 3 – Therapeutics Research in 2017

  • Ex-Vivo Stem Cell Gene Therapy: Approved Treatment for ADA-SCID – Claudio Bordignon, MolMed S.p.A., Italy
  • Approval of a Stem Cell Therapy for Corneal Disease, Holoclar® – Graziella Pellegrini, University of Modena and Reggio Emilia, Italy
  • Gene Therapy for Neurological Disorders: A Promising Novel Treatment for AADC Deficiency – Jodi Cook, Agilis Biotherapeutics LLC, USA
  • Development of Therapy for GNE Myopathy – Ichizo Nishino, National Center of Neurology and Psychiatry, Japan
  • Patient Engagement in Therapeutics Development – Sangeeta Jethwa, Roche Innovation Centre, Switzerland

Parallel Session 2: New Approaches to Rare Diseases

Track 1 – New Approaches to Rare Diseases – Diagnostics

Track 2– New Approaches to Rare Diseases – Foundational

Track 3– New Approaches to Rare Diseases – Therapeutics

Parallel Session 3: Trends in the Field

Track 1 – Trends in Regulatory and Access

Track 2 – Trends in Patient Advocacy

Track 3 – Trends in Companies