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

Start Over Subjects Rare Diseases -- drug therapy ✖

1. 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

2. 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.

3. FDA oversight

Author(s):: White, Robert S., author
Publication:: [Toronto, Ontario] : Thomson Reuters, 23-December-2019
Subject(s):: Drug Approval
Drug Therapy
Government Regulation
Orphan Drug Production
Rare Diseases -- drug therapy
Humans
United States
United States. Food and Drug Administration.

4. 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