Twice weekly dosing with Sebelipase alfa (Kanuma®) rescues severely ill infants with Wolman disease.

Background: Sebelipase alfa (Kanuma®) is approved for patients with Wolman disease (WD) at a dosage of 3-5 mg/kg once weekly. Survival rates in the second of two clinical trials was greater, despite recruiting more severely ill patients, probably related to higher initial and maximal doses. We aimed to evaluate the effective pharmacokinetics and pharmacodynamics of Sebelipase alfa when administered to patients with severe WD at 5 mg/kg twice weekly, an intensive regimen which was not assessed in the trials.

Perspectives on Emergency Preparedness Among Indigenous Pacific People in Hawaii: A Qualitative Study.

Purpose: This study sought an improved understanding of household emergency preparedness (EP) among Native Hawaiian, Pacific Islander, and Filipino (Indigenous Pacific People [IPP]) parents in Hawaii.

Design: We conducted an exploratory qualitative descriptive study with 60-minute interviews occurring from October 2022 through March 2023. A semi-structured interview guide exploring participant household EP was employed.

Advantages of the use of citrate over acetate as a stabilizer in hemodialysis fluid: A randomized ABC-treat study.

Hemodialysis (HD) with bicarbonate dialysis fluid (DF) requires the presence of an acid to prevent the precipitation of calcium and magnesium carbonate. The most used acid is acetic acid, with it several complications have been described. In a previous work we described the acute changes during an HD session with a DF with citrate instead of acetate.

Advantages of the use of citrate over acetate as a stabilizer in hemodialysis fluid: A randomized ABC-treat study.

Hemodialysis (HD) with bicarbonate dialysis fluid (DF) requires the presence of an acid to prevent the precipitation of calcium and magnesium carbonate. The most used acid is acetic acid, with it several complications have been described. In a previous work, we described the acute changes during an HD session with a DF with citrate instead of acetate.

[Bacterial endogenous endophthalmitis].

Endogenous bacterial endophthalmitis is an uncommon disease, secondary to hematogenous spread of a septic focus. Despite advances in diagnostic and therapeutic methods, it remains a potential cause of blindness. We report a case of a diabetic patient with endogenous endophthalmitis of the right eye secondary to an Escherichia coli bacteremia of urinary origin.