Publications by authors named "A Linhart"
Article Synopsis
- Pegunigalsidase alfa is a new enzyme replacement therapy for Fabry disease, showing a longer half-life and administered every 4 weeks instead of the typical 2-week schedule.
- The BRIGHT study involved 30 adult patients who switched from another ERT to pegunigalsidase alfa, revealing good safety results with mostly mild side effects and no new anti-drug antibodies.
- Although the treatment showed acceptable tolerance, more research is needed due to the small sample size, but it suggests that this 4-week regimen could be a viable new option for managing Fabry disease.
Article Synopsis
- * This study aimed to explore long-term pulmonary perfusion issues in COVID-19 survivors who experienced severe acute respiratory distress syndrome (ARDS) and were treated with ECMO at a specific institution.
- * Out of 80 patients who survived ECMO treatment, only 37 were analyzed; while VTE occurred frequently during acute illness, no ongoing pulmonary perfusion abnormalities were found after an average of 14 months post-ECMO.
Article Synopsis
- The study evaluated the 1-year outcomes of the ECMO-CS trial, which compared immediate ECMO initiation to an early conservative approach in patients with severe cardiogenic shock.
- Results showed no significant difference in all-cause mortality or other critical outcomes between the two strategies after one year.
- However, there was a suggestion that immediate ECMO could be more beneficial for patients with significantly low mean arterial pressure at baseline, indicating a possible targeted use for this treatment strategy.
Fabry disease is a progressive, X-linked lysosomal disorder caused by reduced or absent α-galactosidase A activity due to GLA variants. The effects of migalastat were examined in a cohort of 125 Fabry patients with migalastat-amenable GLA variants in the followME Pathfinders registry (EUPAS20599), an ongoing, prospective, patient-focused registry evaluating outcomes for current Fabry disease treatments. We report annualised estimated glomerular filtration rate (eGFR) and Fabry-associated clinical events (FACEs) in a cohort of patients who had received ≥3 years of migalastat treatment in a real-world setting.
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