Hiroyuki Iwasaki

TL;DR: A galactose derivative synthesized for chemical chaperone therapy of a human neurogenetic disease, β-galactosidosis, resulted in significant enhancement of the enzyme activity in the brain and other tissues and a marked decrease of intracellular substrate storage.

TL;DR: Oral administration of a chaperone compound N‐octyl‐4‐epi‐β‐valienamine to GM1‐gangliosidosis model mice was delivered rapidly to the brain, increased β‐galactosidase activity, decreased ganglioside GM1, and prevented neurological deterioration within a few months.

TL;DR: Screening of β-galactosidase-deficient fibroblasts for possible chemical chaperone therapy using N-octyl-4-epi-β-valienamine (NOEV) in patients with GM1-gangliosidosis and Morquio B disease estimated that the NOEV chaper one therapy will be effective in 20–40% of the patients.

TL;DR: The feasibility of TDM‐621, the synthetic infectious agent‐free peptides, was tested in hemostasis of the bleeding after endoscopic treatments of the gastric tumors.

TL;DR: The possibility of skull base metastasis from FTC should be considered in patients with clinical symptoms of cranial nerve dysfunction and radiological findings of bone destruction, and the results confirmed the presence of metastatic tumors in the skull base.