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National Health and Medical Research Council

Cellular and mitochondrial changes in glutamate-induced HT4 neuronal cell death
611 Tafamidis is another pharmacological chaperone that has gained approval for clinical use, specifically in the treatment of TTR amyloidosis

In the clinical trial of HER2-CAR VSTs for progressive glioblastoma, 8 out of 17 patients experienced clinical benefits, with 1 patient having a partial response lasting over 9 months, and 7 patients maintaining stable disease for 8 weeks to 29 months
