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Sous presse. Epreuves corrigées par l'auteur. Disponible en ligne depuis le mardi 10 mars 2020
Doi : 10.1016/j.therap.2020.02.007
Received : 16 September 2019 ;  accepted : 15 November 2019
Drug repositioning for rare diseases: Knowledge-based success stories

Daniel Scherman a, b, c, d, , Christine Fetro a
a French foundation for rare diseases, 75014 Paris, France 
b Chemical and biological technologies for health unit (UTCBS), CNRS UMR8258, 75006 Paris, France 
c UTCBS, Inserm U1267, 75006 Paris, France 
d Faculté de pharmacie, université Paris Descartes, université de Paris, 75006 Paris, France 

Corresponding author. Fondation maladies rares, 96, rue Didot, 75014 Paris, France.Fondation maladies rares96, rue DidotParis75014France

While more than 7000 rare diseases have been identified, only about 5 percent benefit from a licensed treatment. As the majority of these diseases is life threatening, these facts underscore the need for new drugs. Drug repositioning is an alternative strategy in drug development, which represents an attractive opportunity for rare diseases. Drug repositioning (also called drug repurposing, drug reprofiling or drug re-tasking) consists in identifying for an already approved or investigational drug a new use outside the scope of the original medical indication. Drug repositioning is considered in the field of orphan drugs as being a faster and somehow less costly strategy than traditional new drug development for pharmaceutical companies. While several successful repositioning cases have been discovered by serendipity, most successes straightly derive from the molecular characterization of the concerned disease. This short commentary is mainly dedicated to these rationally-based success stories.

The full text of this article is available in PDF format.

Keywords : Rare diseases, Drug repurposing, Drug repositioning, Progeria, Alkaptonuria, Overgrowth syndrome

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