Office of Orphan Products Development from Food and Drug Administration

 
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2017

  • Prospective Study in Angelman Syndrome
  • Prospective Study in Friedreich Ataxia
  • Prospective Study in Myotonic Dystrophy Type 1 to Determine Biomarkers and Clinical Endpoints
  • Prospective Study in Pregnancy & Lactation Associated Osteoporosis
  • Prospective Study in Sickle Cell Anemia: Determine Biomarkers of Endothelial Function Changes in Chronic Kidney Disease
  • Retrospective Study in Sarcoidosis

2013

  • Phase 2 Study of Esophageal String Test in Diagnosing Eosinophilic Esophagitis