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.
