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Vol. 16, No. 6, 2013
Issue release date: February 2014
Section title:  
Public Health Genomics 2013;16:259-267
(DOI:10.1159/000355929)

New and Evolving Rare Diseases Research Programs at the National Institutes of Health

Groft S.C. · Rubinstein Y.R.
Office of Rare Diseases Research, National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, Md., USA

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Article / Publication Details

First-Page Preview
Abstract of  

Published online: 2/3/2014

Number of Print Pages: 9
Number of Figures: 0
Number of Tables: 1

ISSN: 1662-4246 (Print)
eISSN: 1662-8063 (Online)

For additional information: http://www.karger.com/PHG

Abstract

Research emphasis on rare diseases and orphan products remains a major focus of the research Institutes and Centers of National Institutes of Health (NIH). NIH provides more than USD 31 billion annually in biomedical research and research support. This research is the basis of many of the health advances in rare and common diseases. Numerous efforts and a major emphasis by the public and private sector initiatives have resulted in an increase of interventions and diagnostics for rare diseases. Newer translational research programs provide a more systematic and coordinated approach to rare diseases research and orphan products development. The approach that is offered requires extensive public-private partnerships with the pharmaceutical industry, contract research organizations, philanthropic foundations, medical and scientific advisory boards, patient advocacy groups, the academic research community, research and regulatory scientists, government funding agencies, and the public. Each program is unique and requires lengthy planning and collaborative efforts to reach programmatic goals.


  

Author Contacts

Yaffa R. Rubinstein, PhD
Office of Rare Diseases Research, National Center for Advancing Translational Sciences
National Institutes of Health
6701 Democracy Boulevard, Bethesda, MD 20892 (USA)
E-Mail rubinsty@mail.nih.gov

  

Article Information

Published online: February 3, 2014
Number of Print Pages : 9
Number of Figures : 0, Number of Tables : 1, Number of References : 29

  

Publication Details

Public Health Genomics

Vol. 16, No. 6, Year 2013 (Cover Date: February 2014)

Journal Editor: Brand A.M. (Maastricht)
ISSN: 1662-4246 (Print), eISSN: 1662-8063 (Online)

For additional information: http://www.karger.com/PHG


Article / Publication Details

First-Page Preview
Abstract of  

Published online: 2/3/2014

Number of Print Pages: 9
Number of Figures: 0
Number of Tables: 1

ISSN: 1662-4246 (Print)
eISSN: 1662-8063 (Online)

For additional information: http://www.karger.com/PHG


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Drug Dosage: The authors and the publisher have exerted every effort to ensure that drug selection and dosage set forth in this text are in accord with current recommendations and practice at the time of publication. However, in view of ongoing research, changes in goverment regulations, and the constant flow of information relating to drug therapy and drug reactions, the reader is urged to check the package insert for each drug for any changes in indications and dosage and for added warnings and precautions. This is particularly important when the recommended agent is a new and/or infrequently employed drug.
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