Toya Ohashi

TL;DR: These guidelines are presented for the selection and interpretation of methods for use by investigators who aim to examine macroautophagy and related processes, as well as for reviewers who need to provide realistic and reasonable critiques of papers that are focused on these processes.

TL;DR: Migalastat offers promise as a first-in-class oral monotherapy alternative treatment to intravenous ERT for patients with Fabry disease and amenable mutations.

TL;DR: In this paper, a combination of whole-embryo culture, followed by organ culture, encourages exogenous human mesenchymal stem cells to differentiate and contribute to functional complex structures of the new kidney.

TL;DR: Findings suggest that human mesenchymal stem cells can differentiate into a mature renal structure with the potential to replace lost kidney function.

TL;DR: HSCT showed effectiveness towards brain or heart involvement, when performed before signs of brain atrophy or valvular regurgitation appear, and it is considered HSCT is worthwhile in early stages of the disease for patients with MPS II.