Associated Papers
Treating late-onset Tay Sachs disease: Brain delivery with a dual trojan horse protein.
Osher E et al.
Molecular Therapy. Methods & Clinical Development, 2024
sp2-Iminosugars targeting human lysosomal β-hexosaminidase as pharmacological chaperone candidates for late-onset Tay-Sachs disease.
González-Cuesta M et al.
Journal of Enzyme Inhibition and Medicinal Chemistry, 2022
Effect of cyclic, low dose pyrimethamine treatment in patients with Late Onset Tay Sachs: an open label, extended pilot study.
Osher E et al.
Orphanet Journal of Rare Diseases, 2015
Chaperone therapy for GM2 gangliosidosis: effects of pyrimethamine on β-hexosaminidase activity in Sandhoff fibroblasts.
Chiricozzi E et al.
Molecular Neurobiology, 2014
Pyrimethamine as a potential pharmacological chaperone for late-onset forms of GM2 gangliosidosis.
Maegawa GH et al.
The Journal of Biological Chemistry, 2007
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