TY - JOUR
T1 - Drug Repurposing for Rare Diseases
AU - Roessler, Helen, I
AU - Knoers, Nine V. A. M.
AU - van Haelst, Mieke M.
AU - van Haaften, Gijs
N1 - Publisher Copyright: © 2021 Elsevier Ltd Copyright: Copyright 2021 Elsevier B.V., All rights reserved.
PY - 2021/4
Y1 - 2021/4
N2 - Currently, there are about 7000 identified rare diseases, together affecting 10% of the population. However, fewer than 6% of all rare diseases have an approved treatment option, highlighting their tremendous unmet needs in drug development. The process of repurposing drugs for new indications, compared with the development of novel orphan drugs, is a time-saving and cost-efficient method resulting in higher success rates, which can therefore drastically reduce the risk of drug development for rare diseases. Although drug repurposing is not novel, new strategies have been developed in recent years to do it in a systematic and rational way. Here, we review applied methodologies, recent accomplished progress, and the challenges associated in drug repurposing for rare diseases.
AB - Currently, there are about 7000 identified rare diseases, together affecting 10% of the population. However, fewer than 6% of all rare diseases have an approved treatment option, highlighting their tremendous unmet needs in drug development. The process of repurposing drugs for new indications, compared with the development of novel orphan drugs, is a time-saving and cost-efficient method resulting in higher success rates, which can therefore drastically reduce the risk of drug development for rare diseases. Although drug repurposing is not novel, new strategies have been developed in recent years to do it in a systematic and rational way. Here, we review applied methodologies, recent accomplished progress, and the challenges associated in drug repurposing for rare diseases.
KW - drug discovery
KW - drug repositioning
KW - drug repurposing
KW - genetic diseases
KW - rare diseases
UR - http://www.scopus.com/inward/record.url?scp=85100542202&partnerID=8YFLogxK
U2 - https://doi.org/10.1016/j.tips.2021.01.003
DO - https://doi.org/10.1016/j.tips.2021.01.003
M3 - Review article
C2 - 33563480
SN - 0165-6147
VL - 42
SP - 255
EP - 267
JO - Trends in pharmacological sciences
JF - Trends in pharmacological sciences
IS - 4
ER -