Drug Repurposing for Rare Diseases
February 2021
in “
Trends in Pharmacological Sciences
”
Studysummary This review discusses drug repurposing strategies for rare diseases, highlighting methodologies, achievements, and challenges, but reports no new clinical results.
Our plain-language summary. Not medical advice or a treatment recommendation. Consult a qualified healthcare professional before changing treatment. Full disclaimer
The document discussed the growing interest in drug repurposing as a method to address the unmet needs of rare diseases, which affected 10% of the population with only 6% having approved treatments. Advances in DNA sequencing and big data analysis facilitated the identification of molecular drivers of these diseases, raising hopes for new treatments. Drug repurposing was highlighted as a cost-effective and time-saving alternative to traditional drug development, with systematic approaches leading to promising candidate drugs in clinical trials or already approved. Despite challenges, this approach offered a higher success rate and reduced risk in developing treatments for rare diseases.
