Balancing benefits and burdens: a systematic review on ethical and social dimensions of gene and cell therapies for hereditary blood diseases.

Background: Sickle cell disease (SCD) and Diamond-Blackfan anemia syndrome (DBAS) are two hereditary blood diseases that present significant challenges to patients, their caregivers, and the healthcare system. Both conditions cause severe health complications and have limited treatment options, leaving many individuals without access to curative therapies like hematopoietic stem cell transplantation. Recent advancements in gene and cell therapies offer the potential for a new curative option, marking a pivotal shift in the management of these debilitating diseases.

Deep representation learning for clustering longitudinal survival data from electronic health records.

Precision medicine requires accurate identification of clinically relevant patient subgroups. Electronic health records provide major opportunities for leveraging machine learning approaches to uncover novel patient subgroups. However, many existing approaches fail to adequately capture complex interactions between diagnosis trajectories and disease-relevant risk events, leading to subgroups that can still display great heterogeneity in event risk and underlying molecular mechanisms.

Number, treatment, and mortality of paediatric pelvic ring fractures in two level 1 trauma centres in the Netherlands : a multicentre retrospective cohort study.

Aims: Paediatric pelvic ring fractures are rare but severe injuries, presenting significant treatment challenges. This study aimed to analyze patient characteristics and explore trends in incidence, treatment methods, and mortality associated with these injuries.

Methods: This multicentre, retrospective cohort study analyzed paediatric patients (aged ≤ 18 years) with pelvic ring fractures treated between 2001 and 2021 at two level 1 trauma centres.

Survivin in cardiovascular diseases and its therapeutic potential.

Aberrant changes in cell behaviors, such as proliferation, apoptosis, and migration, are some of the contributing factors to the development of various cardiovascular diseases (CVDs) and pathologies, including atherosclerosis, neointimal hyperplasia, and heart failure. In recent years, numerous studies have identified survivin, a key player in the anti-apoptotic pathway, to be extensively involved in modulating cellular functioning in cancer, with many reaching clinical trials. Though seemingly different, CVDs and cancer share abundant similarities regarding abnormal cell modifications and behaviors.