A Systematic Review on Safety and Efficacy of Migalastat for the treatment of Fabry's Disease.

Introduction: Fabry's disease (FD) is a genetic lysosomal storage disorder characterized by α-galactosidase A (α-Gal A) lost/reduced activity. We aim to systematically assess the safety and efficacy of Migalastat, an oral pharmacological chaperone, that has been approved for the treatment of FD in patients with amenable mutations.

Methods: We conducted literature search following the PRISMA guidelines in major databases up to 4 February 2024, for studies that assessed the clinical outcomes of migalastat in patients with FD.

The low density and magnetization of a massive galaxy halo exposed by a fast radio burst.

Present-day galaxies are surrounded by cool and enriched halo gas extending for hundreds of kiloparsecs. This halo gas is thought to be the dominant reservoir of material available to fuel future star formation, but direct constraints on its mass and physical properties have been difficult to obtain. We report the detection of a fast radio burst (FRB 181112), localized with arcsecond precision, that passes through the halo of a foreground galaxy.