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 New Castle Ottawa Scale was used to evaluate the quality of the included studies. RESULTS: A total of 2141 records were identified through database searches and register searches, amongst which 26 records were screened, and 12 of these were excluded. The remaining 14 reports were sought for retrieval. The 12 retrieved articles were assessed for eligibility and their quality was assessed after their inclusion. Amongst the included studies, 5 were of high quality, 6 were of medium quality, and 1 was of low quality. CONCLUSION: Migalastat showed varied effects on enzyme activity and substrate levels, with gender-specific differences noted in GL-3 substrate activity and eGFR. Overall, it improved cardiac and renal outcomes similarly to enzyme replacement therapy, with a comparable safety profile.

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. Analysis of the burst shows that the halo gas has low net magnetization and turbulence. Our results imply predominantly diffuse gas in massive galactic halos, even those hosting active supermassive black holes, contrary to some previous results.