Journal of Genetic Syndromes & Gene Therapy
Open Access

5^th Annual Congress on Rare Diseases and Orphan Drugs

August 29-30, 2018 | Boston, USA

Scientific Tracks Abstracts

• Re-dosing AAV gene therapy vectors - the elephant in the room

  Kei Kishimoto

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• Latin America calls members of congress to legislate on rare diseases

  Fernando Ferrer

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• Helping patients cope with, and recover from the effects of living with a rare disease: A patients perspective

  Mark Landiak

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• Overview of rare disease funding at NIH

  Jason Wan

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• A comparative analysis of demographic information among 12 neural intractable diseases in a national registry of a rare disease in Japan

  Yoko Sato and Yasuhiro Kanatani

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• Macrophage activation syndrome and acquired hemophagocytic lymphohistiocytosis in adults: A Philadelphia cohort

  Irene Tan

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• Sarcoidosis: The most common rare disease

  Mark Landiak

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• How to educate others about your rare disease

  Dana Mauro

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