The term “pharmacological chaperone” was introduced 20 years ago. Since then the
approach with this type of drug has been proposed for several diseases, lysosomal storage …

The treatment landscape for lysosomal storage disorders (LSDs) is rapidly evolving. An
increase in the number of preclinical and clinical studies in the last decade has …

Fabry disease (FD) is a rare metabolic disorder of glycosphingolipid storage caused by
mutations in the GLA gene encoding lysosomal hydrolase α‐galactosidase A (α‐gal A) …

Plant extracts are one of the best possible sources of bioactive molecules, and are being
used globally as an antioxidants and natural antimicrobial compounds. In current study …

Fabry disease is caused by a deficiency of lysosomal alpha galactosidase and has a very
large genotypic and phenotypic spectrum. Some patients who carry hypomorphic mutations …

Fabry disease is a lysosomal storage disease caused by mutations in the GLA gene that
encodes alpha-galactosidase (AGAL). The disease causes abnormal globotriaosylceramide …

Enzyme replacement therapy is the only therapeutic option for Fabry patients with
completely absent AGAL activity. However, the treatment has side effects, is costly, and …

Lysosomal storage disorders (LSD) are a group of heterogeneous diseases caused by
compromised enzyme function leading to multiple organ failure. Therapeutic approaches …

In Gaucher disease (GD), mutant lysosomal acid β‐glucocerebrosidase fails to properly
hydrolyze its substrate, glucosylceramide, which accumulates in the lysosomes. Due to its …

Fabry disease (FD) is a progressive, X-linked inherited disorder of glycosphingolipid
metabolism due to deficient or absent lysosomal α-galactosidase A (α-Gal A) activity which …