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.

Systematic review of MRI safety literature in relation to radiofrequency thermal injury prevention.

Introduction: Magnetic resonance imaging (MRI) is a rapidly evolving modality, generally considered safe due to lack of ionising radiation. While MRI technology and techniques are improving, many of the safety concerns remain the same as when first established. Patient thermal injuries are the most frequently reported adverse event, accounting for 59% of MRI incidents to the Food and Drug Administration (FDA).

Genetic Transformation of Cryptococcus Species with Agrobacterium Transfer DNA.

Transformation of foreign DNA into Cryptococcus species is a powerful tool for exploring gene functions in these human pathogens. Agrobacterium tumefaciens-mediated transformation (AtMT) has been used for the stable introduction of exogenous DNA into Cryptococcus for over two decades, being particularly impactful for insertional mutagenesis screens to discover new genes involved in fungal biology. A detailed protocol to conduct this transformation method is provided in the chapter.

Developmental transcriptomic patterns can be altered by transgenic overexpression of Uty.

The genetic material encoded on X and Y chromosomes provides the foundation by which biological sex differences are established. Epigenetic regulators expressed on these sex chromosomes, including Kdm6a (Utx), Kdm5c, and Ddx3x have far-reaching impacts on transcriptional control of phenotypic sex differences. Although the functionality of UTY (Kdm6c, the Y-linked homologue of UTX), has been supported by more recent studies, its role in developmental sex differences is not understood.

Impact of Race and Area Deprivation on Triple-Negative Metastatic Breast Cancer Outcomes.

Objectives: To describe area deprivation, anxiety, depression, relative dose intensity of first-line metastatic breast cancer (MBC) treatment, and survival in Black and White women who had died from triple-negative MBC, including interaction analysis.

Sample & Setting: This cohort study drew from a database of women who had died from MBC (N = 53).

Methods & Variables: Descriptive statistics, independent t tests, analysis of variance, and Mann-Whitney U tests were used, and effect sizes were calculated.