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REPORT

Medicines in Development for Rare Diseases 2016 Report

America's biopharmaceutical research companies are developing 566 medicines for patients with rare diseases.

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REPORT

List of 2016 Medicines in Development for Rare Diseases

This report outlines the list of Medicines in Development for rare diseases and includes the product name, sponsor, official FDA designation and development status.

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REPORT

2005-2015: A Decade of Innovation In Rare Diseases

There is an urgent need for continued research to map the genes responsible for rare diseases, identify genetic and other biologic anomalies that contribute to these conditions, and understand the natural history of rare diseases to inform the development of potential therapies.