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PHRMA Report – More than 300 Medicines Now In Development to Treat Rare Diseases

Press release: “There are more than 300 medicines currently in development to treat or prevent hundreds of rare diseases, according to a new report released today by the Pharmaceutical Research and Manufacturers of America (PhRMA). Genetic Alliance and the National Organization for Rare Disorders (NORD) joined PhRMA in releasing the report.”

  • Read the Report (32 pages, PDF)
  • Read the Backgrounder, Selected Examples of Orphan Drugs in Development for Rare Diseases
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