Designing clinical trials for rare diseases: unique challenges and opportunities
Chiara Pizzamiglio, Hilary J. Vernon, Michael G. Hanna, Robert D. S. Pitceathly
Topics & Concepts
Clinical trialOrphan drugDrug developmentMedicineIntensive care medicineClinical study designMedical physicsDrugBioinformaticsPathologyPharmacologyBiologyLysosomal Storage Disorders ResearchGenomics and Rare DiseasesNeurogenetic and Muscular Disorders Research