# Drug Repurposing and Lysosomal Storage Disorders: A Trick to Treat

## TL;DR

This paper explores using existing drugs for new treatments of rare lysosomal storage disorders, aiming to speed up therapy development.

## Contribution

The study provides a systematic analysis of drug repurposing potential for lysosomal storage disorders with a focus on mutation-specific approaches.

## Key findings

- Drug repurposing can reduce costs and time in developing therapies for lysosomal storage disorders.
- Mutation-specific drug strategies are essential for effective treatment of lysosomal storage disorders.
- Careful evaluation of drug efficacy and toxicity is necessary for successful repurposing in rare diseases.

## Abstract

Rare diseases, or orphan diseases, are defined as diseases affecting a small number of people compared to the general population. Among these, we find lysosomal storage disorders (LSDs), a cluster of rare metabolic diseases characterized by enzyme mutations causing abnormal glycolipid storage. Drug repositioning involves repurposing existing approved drugs for new therapeutic applications, offering advantages in cost, time savings, and a lower risk of failure. We present a comprehensive analysis of existing drugs, their repurposing potential, and their clinical implications in the context of LSDs, highlighting the necessity of mutation-specific approaches. Our review systematically explores the landscape of drug repositioning as a means to enhance LSDs therapies. The findings advocate for the strategic repositioning of drugs, accentuating its role in expediting the discovery of effective treatments. We conclude that drug repurposing represents a viable pathway for accelerating therapeutic discovery for LSDs, emphasizing the need for the careful evaluation of drug efficacy and toxicity in disease-specific contexts.