Drug Repurposing and Lysosomal Storage Disorders: A Trick to Treat
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...
Main Authors: | , , , , |
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Format: | Article |
Language: | English |
Published: |
MDPI AG
2024-02-01
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Series: | Genes |
Subjects: | |
Online Access: | https://www.mdpi.com/2073-4425/15/3/290 |