Subjects: Rare Diseases -- drug therapy - Digital Collections - National Library of Medicine Search Results

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NLM Digital Collections

Start Over Subjects Rare Diseases -- drug therapy

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 -- standards
Drugs, Investigational
Rare Diseases -- drug therapy
Metabolism, Inborn Errors -- drug therapy
United States
United States. Department of Health and Human Services
United States. Food and Drug Administration

2. Drug development: FDA's priority review voucher programs : report to Congressional committees

Publication:: Washington, DC : United States Government Accountability Office, January 2020
Subject(s):: Drug Approval -- organization & administration
Drug Development -- organization & administration
Rare Diseases -- drug therapy
United States
United States. Food and Drug Administration

3. Rare diseases: considerations for the development of drugs and biological products : guidance for industry

Publication:: Silver Spring, MD : Center for Drug Evaluation and Research, December 2023
Subject(s):: Biological Products
Drug Development
Drug Industry
Government Regulation
Rare Diseases -- drug therapy
United States