Drug repositioning for rare diseases: Knowledge-based success stories - Université Paris Cité Accéder directement au contenu
Article Dans Une Revue Thérapie Année : 2020

Drug repositioning for rare diseases: Knowledge-based success stories

Résumé

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.
Fichier principal
Vignette du fichier
S0040595720300123.pdf (827.66 Ko) Télécharger le fichier
Origine : Fichiers produits par l'(les) auteur(s)

Dates et versions

hal-03490261 , version 1 (20-05-2022)

Licence

Paternité - Pas d'utilisation commerciale

Identifiants

Citer

Daniel Scherman, Christine Fetro. Drug repositioning for rare diseases: Knowledge-based success stories. Thérapie, 2020, 75, pp.161 - 167. ⟨10.1016/j.therap.2020.02.007⟩. ⟨hal-03490261⟩
23 Consultations
180 Téléchargements

Altmetric

Partager

Gmail Facebook X LinkedIn More