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Erschienen in: Wiener Medizinische Wochenschrift 9-10/2017

01.06.2017 | main topic

Orphan diseases: state of the drug discovery art

verfasst von: Claude-Henry Volmar, PhD, Claes Wahlestedt, MD, PhD, Shaun P. Brothers, PhD

Erschienen in: Wiener Medizinische Wochenschrift | Ausgabe 9-10/2017

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Summary

Since 1983 more than 300 drugs have been developed and approved for orphan diseases. However, considering the development of novel diagnosis tools, the number of rare diseases vastly outpaces therapeutic discovery. Academic centers and nonprofit institutes are now at the forefront of rare disease R&D, partnering with pharmaceutical companies when academic researchers discover novel drugs or targets for specific diseases, thus reducing the failure risk and cost for pharmaceutical companies. Considerable progress has occurred in the art of orphan drug discovery, and a symbiotic relationship now exists between pharmaceutical industry, academia, and philanthropists that provides a useful framework for orphan disease therapeutic discovery. Here, the current state-of-the-art of drug discovery for orphan diseases is reviewed. Current technological approaches and challenges for drug discovery are considered, some of which can present somewhat unique challenges and opportunities in orphan diseases, including the potential for personalized medicine, gene therapy, and phenotypic screening.
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Metadaten
Titel
Orphan diseases: state of the drug discovery art
verfasst von
Claude-Henry Volmar, PhD
Claes Wahlestedt, MD, PhD
Shaun P. Brothers, PhD
Publikationsdatum
01.06.2017
Verlag
Springer Vienna
Erschienen in
Wiener Medizinische Wochenschrift / Ausgabe 9-10/2017
Print ISSN: 0043-5341
Elektronische ISSN: 1563-258X
DOI
https://doi.org/10.1007/s10354-015-0423-0

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