Book contents
- Frontmatter
- Contents
- Contributors
- Preface
- SECTION I INFRASTRUCTURE
- 1 The U.S. Food and Drug Administration and the regulation of small molecules for orphan diseases
- 2 The Office of Rare Diseases Research: Serving a coordinating function at the National Institutes of Health
- 3 Introduction to pharmacokinetics and pharmacodynamics
- SECTION II COFACTORS
- SECTION III UTILIZATION OF ALTERNATIVE PATHWAYS
- SECTION IV METAL ION THERAPY
- Index
- Plates section
- References
2 - The Office of Rare Diseases Research: Serving a coordinating function at the National Institutes of Health
from SECTION I - INFRASTRUCTURE
Published online by Cambridge University Press: 17 November 2010
Edited by
Book contents
- Frontmatter
- Contents
- Contributors
- Preface
- SECTION I INFRASTRUCTURE
- 1 The U.S. Food and Drug Administration and the regulation of small molecules for orphan diseases
- 2 The Office of Rare Diseases Research: Serving a coordinating function at the National Institutes of Health
- 3 Introduction to pharmacokinetics and pharmacodynamics
- SECTION II COFACTORS
- SECTION III UTILIZATION OF ALTERNATIVE PATHWAYS
- SECTION IV METAL ION THERAPY
- Index
- Plates section
- References
- Type
- Chapter
- Information
- Small Molecule Therapy for Genetic Disease , pp. 19 - 34Publisher: Cambridge University PressPrint publication year: 2010
References
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: Appendices – Volume I, Publication No. HRP-0907249, 10–11, Springfield, VA: National Technical Information Services; April 1989Google Scholar
, NIH News, May 19, 2008. Available at: http://www.nih.gov/news/health/may2008/nhgri-19.htm
, , , , , , , , . Rare diseases clinical research network: Clinical research for rare disease: Opportunities, challenges, and solutions. Mol Genet Metab. 2009;96(1):20–26CrossRefGoogle ScholarPubMed
, , , . Rare childhood diseases: How should we respond?Arch Dis Child. 2008;93(12):1071–1074CrossRefGoogle ScholarPubMed