Therapeutic Role of Pharmacological Chaperones in Lysosomal Storage Disorders: A Review of the Evidence and Informed Approach to Reclassification

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 demonstrated that pharmacological chaperones are a feasible alternative to enzyme replacement therapy (ERT) for individuals with LS...

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Detalles Bibliográficos
Autores principales: Keyzor, Ian, Shohet, Simon, Castelli, Jeff, Sitaraman, Sheela, Veleva-Rotse, Biliana, Weimer, Jill M., Fox, Brian, Willer, Tobias, Tuske, Steve, Crathorne, Louise, Belzar, Klara J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Systematic Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10452329/
https://www.ncbi.nlm.nih.gov/pubmed/37627292
http://dx.doi.org/10.3390/biom13081227

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