Tissue chips to aid drug development and modeling for rare diseases
نویسندگان
چکیده
منابع مشابه
A methodological framework for drug development in rare diseases
INTRODUCTION Developing orphan drugs is challenging because of their severity and the requisite for effective drugs. The small number of patients does not allow conducting adequately powered randomized controlled trials (RCTs). There is a need to develop high quality, ethically investigated, and appropriately authorized medicines, without subjecting patients to unnecessary trials. AIMS AND OB...
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BACKGROUND We studied to what extent the level of scientific knowledge on exceptionally rare metabolic inherited diseases and their potential orphan medicinal products is associated with sponsors deciding to apply for an orphan designation at the US Food and Drug Administration (FDA) or the European Medicines Agency (EMA). METHODS All metabolic diseases with a genetic cause and prevalence of ...
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ژورنال
عنوان ژورنال: Expert Opinion on Orphan Drugs
سال: 2016
ISSN: 2167-8707
DOI: 10.1080/21678707.2016.1244479