Journal of Genetic Syndromes & Gene Therapy
Open Access

3^rd Annual Congress on Rare Diseases and Orphan Drugs

October 30-November 01, 2017 San Antonio, USA

Keynote

• Rare diseases and orphan drugs: Passion and compassion or growing market with career opportunities for scientists and technologists?

  Harsha K Rajasimha

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• Speeding up access to medicines for patients with unmet medical need: Integrating evidence and regulatory pathways

  Stella Blackburn

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• Mitochondrial bioenergetics in porphyria: Studies in peripheral blood cells

  Ashwani K Singal

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• Pediatric rare disease enrollment case study in Latin America

  Sara G Tylosky

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• OP-101: A novel therapy for treatment of childhood cerebral adrenoleukodystrophy

  Sujatha Kannan

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• Lifting the burden of rare disease by providing access to next generation sequencing

  Romina Ortiz

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• Consumer directed precision health - convergence of multi- omics, environment, life style and behaviors

  Praduman Jain

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