Articles: function.
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Randomized Controlled Trial
Trial of N-Acetyl-l-Leucine in Niemann-Pick Disease Type C.
Niemann-Pick disease type C is a rare lysosomal storage disorder. We evaluated the safety and efficacy of N-acetyl-l-leucine (NALL), an agent that potentially ameliorates lysosomal and metabolic dysfunction, for the treatment of Niemann-Pick disease type C. ⋯ Among patients with Niemann-Pick disease type C, treatment with NALL for 12 weeks led to better neurologic status than placebo. A longer period is needed to determine the long-term effects of this agent in patients with Niemann-Pick disease type C. (Funded by IntraBio; ClinicalTrials.gov number, NCT05163288; EudraCT number, 2021-005356-10.).
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Patients undergoing cardiac surgery are at high risk to develop cardiac surgery-associated acute kidney injury (CS-AKI) postoperatively. CS-AKI is associated with an increased risk for persistent renal dysfunction, morbidity and mortality. This review summarizes the epidemiology and pathophysiology of CS-AKI, as well as current treatment and prevention strategies. ⋯ Based on the described novel approaches for early detection, prevention and management of CS-AKI, a precision-medicine approach should be implemented in order to prevent the development of AKI in patients undergoing cardiac surgery.
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Curr Opin Crit Care · Feb 2024
ReviewSetting positive end-expiratory pressure: role in diaphragm-protective ventilation.
With mechanical ventilation, positive end-expiratory pressure (PEEP) is applied to improve oxygenation and lung homogeneity. However, PEEP setting has been hypothesized to contribute to critical illness associated diaphragm dysfunction via several mechanisms. Here, we discuss the impact of PEEP on diaphragm function, activity and geometry. ⋯ Setting PEEP could play an important role in both lung and diaphragm protective ventilation. Both high and low PEEP levels could potentially introduce or exacerbate diaphragm myotrauma. Today, the impact of PEEP setting on diaphragm structure and function is in its infancy, and clinical implications are largely unknown.