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References from Discovery of allosteric regulators with clinical potential to stabilize alpha-L-iduronidase in mucopolysaccharidosis type I. Local targets link to admitted publications; unresolved targets remain external evidence.
The natural history of MPS I: global perspectives from the MPS I Registry
10.1038/gim.2014.25 · 2014 · External reference
Mucopolysaccharidosis I, II, and VI: Brief review and guidelines for treatment
10.1590/s1415-47572010005000093 · 2010 · External reference
Mucopolysaccharidosis Type I: A Review of the Natural History and Molecular Pathology
10.3390/cells9081838 · 2020 · External reference
Epidemiology of Mucopolysaccharidoses Update
2021 · External reference
Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure
10.1186/1750-1172-6-55 · 2011 · External reference
Enzyme replacement therapy: efficacy and limitations
10.1186/s13052-018-0562-1 · 2018 · External reference
Enzyme replacement therapy with laronidase (Aldurazyme (®)) for treating mucopolysaccharidosis type I
2019 · External reference
Effect of anti-laronidase antibodies on efficacy and safety of laronidase enzyme replacement therapy for MPS I: A comprehensive meta-analysis of pooled data from multiple studies
10.1016/j.ymgme.2016.02.006 · 2016 · External reference
Enzyme replacement therapies: what is the best option?
10.15171/bi.2018.17 · 2018 · External reference
Therapeutic Options for Mucopolysaccharidoses: Current and Emerging Treatments
10.1007/s40265-019-01147-4 · 2019 · External reference
Treatment of Fabry’s Disease with the Pharmacologic Chaperone Migalastat
10.1056/nejmoa1510198 · 2016 · External reference
Chaperone Therapy in Fabry Disease
10.3390/ijms23031887 · 2022 · External reference
Ambroxol chaperone therapy for neuronopathic Gaucher disease: A pilot study
10.1002/acn3.292 · 2016 · External reference
Antigen stability controls antigen presentation
10.1074/jbc.m405738200 · 2004 · External reference
Pharmacological Chaperone Therapy: Preclinical Development, Clinical Translation, and Prospects for the Treatment of Lysosomal Storage Disorders
10.1038/mt.2015.62 · 2015 · External reference
The pharmacological chaperone N-butyldeoxynojirimycin enhances enzyme replacement therapy in Pompe disease fibroblasts
10.1038/mt.2009.53 · 2009 · External reference
The pharmacological chaperone AT2220 increases recombinant human acid α-glucosidase uptake and glycogen reduction in a mouse model of Pompe disease
10.1371/journal.pone.0040776 · 2012 · External reference
Synergy between the pharmacological chaperone 1-deoxygalactonojirimycin and the human recombinant alpha-galactosidase A in cultured fibroblasts from patients with Fabry disease
10.1007/s10545-011-9424-3 · 2012 · External reference
Unresolved reference
External reference
Human α-L-iduronidase uses its own N-glycan as a substrate-binding and catalytic module
10.1073/pnas.1306939110 · 2013 · External reference
Binding site detection and druggability index from first principles
10.1021/jm801385d · 2009 · External reference
Molecular Simulations with Solvent Competition Quantify Water Displaceability and Provide Accurate Interaction Maps of Protein Binding Sites
10.1021/jm5010418 · 2014 · External reference
Extracting Atomic Contributions to Binding Free Energy Using Molecular Dynamics Simulations with Mixed Solvents (MDmix)
2022 · External reference
Unresolved reference
External reference
rDock: A Fast, Versatile and Open Source Program for Docking Ligands to Proteins and Nucleic Acids
10.1371/journal.pcbi.1003571 · 2014 · External reference
The use of differential scanning fluorimetry to detect ligand interactions that promote protein stability
10.1038/nprot.2007.321 · 2007 · External reference
Unresolved reference
External reference
Biochemical characterization of fluorescent-labeled recombinant human alpha-L-iduronidase in vitro
10.1002/bab.52 · 2011 · External reference
Evaluating virtual screening methods: good and bad metrics for the "early recognition" problem
10.1021/ci600426e · 2007 · External reference
Hit identification and optimization in virtual screening: practical recommendations based on a critical literature analysis
10.1021/jm301916b · 2013 · External reference
