Pivotal trial characteristics and types of endpoints used to support Food and Drug Administration rare disease drug approvals between 2013 and 2022.

Background/aims: Rare disease drug development faces unique challenges, such as genotypic and phenotypic heterogeneity within small patient populations and a lack of established outcome measures for conditions without previously successful drug development programs. These challenges complicate the process of selecting the appropriate trial endpoints and conducting clinical trials in rare diseases. In this descriptive study, we examined novel drug approvals for non-oncologic rare diseases by the U.

Risk factors for lymphatic leakage following radical cystectomy and pelvic lymph node dissection in patients with muscle-invasive bladder cancer.

Background: Lymphatic leakage is a common complication after radical cystectomy and pelvic lymph node dissection (PLND) for muscle-invasive bladder cancer (MIBC).This study aimed to investigate the risk factors contributing to postoperative lymphatic leakage in patients with MIBC.

Materials And Methods: A total of 534 patients undergoing radical cystectomy and PLND were enrolled in the retrospective study at Peking University Third Hospital from January 2010 to July 2023.

Size-controlled assembly of phase separated protein condensates with interfacial protein cages.

Phase separation of specific proteins into liquid-like condensates is a key mechanism for forming membrane-less organelles, which organize diverse cellular processes in space and time. These protein condensates hold immense potential as biomaterials capable of containing specific sets of biomolecules with high densities and dynamic liquid properties. Despite their appeal, methods to manipulate protein condensate materials remain largely unexplored.

Comparing haploidentical transplantation with post-transplantation cyclophosphamide and umbilical cord blood transplantation using targeted busulfan in children and adolescents with hematologic malignancies.

Purpose: This study compared the outcomes of haploidentical-related donor (HRD) and umbilical cord blood (UCB) hematopoietic stem cell transplantation (HSCT) in pediatric patients with hematologic malignancies.

Methods: Data on patients who underwent HRD HSCT with post-transplant cyclophosphamide (n = 41) and UCB HSCT (n = 24) after targeted busulfan-based myeloablative conditioning with intensive pharmacokinetic monitoring between 2009 and 2018 were retrospectively analyzed.

Results: The median follow-up durations in the HRD and UCB groups were 7.

Effect of epinephrine administration interval on cerebral perfusion in a porcine cardiac arrest model.

Objectives: The recommended epinephrine administration interval during CPR is between 3 and 5 min. However, the optimal interval for improving cerebral perfusion remains controversial. This study aimed to evaluate the effects of epinephrine administration interval of 3 min or 5 min on cerebral perfusion pressure (CEPP) and cortical cerebral blood flow (CCBF) in a porcine cardiac arrest model.