Journal of Genetic Syndromes & Gene Therapy
Open Access

8^th World Congress on Rare Diseases and Orphan Drugs & Clinical Trials & Regulatory Affairs

November 21-22, 2018 | Paris, France

Scientific Tracks Abstracts

• Reimbursement dynamics and market access hurdles for orphan drugs in the EU5

  Akash Saini

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• Specific clinical and evolutionary aspects of adenosine deaminase 2 deficiency (DADA2)

  Djamel Eddine Ouail,Faycal Bouali, Djamila Si Ahmed,Hassina Ahmane, Meriem Tebbani

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• Orphan medicines and paediatric drug development

  Mette Due Theilade Thomsen

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• Real world data in the new era of clinical research

  Elena Tenti, MSc, PharmD

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• Primary cutaneous actinomycosis: A first case report from Kurdistan- Iraq

  Yassob Sulaiman

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• Strategy of attention to rare diseases in Catalonia

  Roser Francisco,Pilar Magrinya, Josep Torrent,Alex Guarga, Cristina Mallol and Cristina Nadal

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• The positive effect of SKD plus IOT and HBO2T in the treatment of cancer

  Sorush Niknamian,Somayeh Zaminpira

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• The evolving landscape of pre-approval access to orphan drugs

  Stuart Bell

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