1. Slowly progressive, low-prevalence rare diseases with substrate deposition that result from single enzyme defects: providing evidence of effectiveness for replacement or corrective therapies Publication: Silver Spring, MD : Center for Drug Evaluation and Research, March 2020 Subject(s): Drug Development -- standardsDrugs, InvestigationalRare Diseases -- drug therapyMetabolism, Inborn Errors -- drug therapyUnited StatesUnited States. Department of Health and Human Services.United States. Food and Drug Administration.
2. FDA oversight Author(s): White, Robert S., author Publication: [Toronto, Ontario] : Thomson Reuters, 23-December-2019 Subject(s): Drug ApprovalDrug TherapyGovernment RegulationOrphan Drug ProductionRare Diseases -- drug therapyHumansUnited StatesUnited States. Food and Drug Administration.
