The Pharmaceutical Industry’s Focus on Rare Diseases

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This edition of the Genetic Technology TOE depicts life sciences trends across rare diseases. Since rare diseases affect only a small portion of the worldwide population; drug discovery, development, and optimization are difficult for such diseases. While only some companies are focusing in this regard, some large pharmaceutical companies are making outstanding advances in drug research and development. This deliverable profiles innovations of four pharmaceutical companies working on new solutions for uncommon and underserved medical conditions. The corresponding clinical trials scenario for Gaucher's disease is also depicted.The Genetic Technology TechVision Opportunity Engine (TOE) presents the latest R&D de

Table of Contents

The Pharmaceutical Industry’s Focus on Rare DiseasesRecent Advances Across Rare DiseasesAdvancing Protein-based Therapy NeoGAA for Pompe’s DiseaseReshaping Business Models to Accelerate Clinical StagesEnhancing Protein Replacement Therapy to Treat Rare DiseasesAccelerating Clinical Trials Pace by Reducing Cycle TimesClinical Trial Analysis and Industry InteractionsClinical Trial Analysis for Gaucher’s DiseaseIndustry Interactions

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