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

Keynote

• Patient-directed insights on the evolution of treating rare genetic diseases

  Mattie Robinson

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• Three-day trial of n-carbamylglutamate improves nitrogen metabolism and decreases ammonia in inherited hyperammonemias and healthy adults

  Leslie Atley,GT Berry, SE McCandless, M Yudkoff,M Tuchman N Ah Mew

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