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2015, ISBN 1621821374, xix, 388 p., 12 unnumbered p.s of plates
.... It recounts the now century long effort of small groups of physicians and scientists to take on some of these genetic diseases... 
Genetic disorders in children | MEDICAL / Diseases | Medical genetics | SCIENCE / Life Sciences / Genetics & Genomics | Genetic Diseases, Inborn | MEDICAL / Pediatrics | Rare Diseases | Research | Child
Book
The New England journal of medicine, ISSN 1533-4406, 10/2019, Volume 381, Issue 17, pp. 1644 - 1652
Journal Article
2013, 1, ISBN 9781906093525, vi, 272
This book presents some of the latest developments in the world of rare disease entrepreneurship from a global group of experts... 
Rare diseases | Treatment | Research | Drug development | Social entrepreneurship | Corporate Social Responsibility & Business Ethics | Environment & Business | Orphan Drug Production | Europe | Entrepreneurship | Organization & administration | Drug therapy | Methods
Book
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Clinical therapeutics, ISSN 0149-2918, 07/2012, Volume 34, Issue 7, pp. 1487 - 1504
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British journal of clinical pharmacology, ISSN 0306-5251, 11/2017, Volume 83, Issue 11, pp. 2386 - 2397
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