Lentivirus-mediated gene therapy for Fabry disease: 5-year End-of-Study results from the Canadian FACTs trial.

Background: Fabry disease is an X-linked lysosomal storage disorder due to a deficiency of α-galactosidase A (α-gal A) activity. Our goal was to correct the enzyme deficiency in Fabry patients by transferring the cDNA for α-gal A into their CD34+ hematopoietic stem/progenitor cells (HSPCs). Overexpression of α-gal A leads to secretion of the hydrolase; which can be taken up and used by uncorrected bystander cells.

Factors associated with patients' demand for low-value care: a scoping review.

Background: Low-value care is unnecessary care that contributes to inefficient use of health resources and constitutes a considerable proportion of healthcare expenditures worldwide. Factors contributing to patients' demand for low-value care have often been overlooked and are dispersed in the literature. Therefore, the current study aimed to systematically summarize factors associated with patients' demand for low-value care.

Ngā Māuiui Kai: creating Indigenous Māori terms for eating disorders in Aotearoa New Zealand.

Background: Māori (the Indigenous people of Aotearoa New Zealand) report higher rates of eating disorders than non-Māori, but access treatment at lower rates. Diagnostic terms lacking in cultural relevance likely contribute to Māori exclusion in eating disorder spaces. Developing terms in te reo Māori (the Māori language) presents an opportunity to challenge eating disorder stereotypes and increase cultural safety in the eating disorder workforce.

A crustal Pb isotope map of southeastern Australia.

Lead isotopes are a powerful geochemical tracer and a popular tool applied across a broad range of scientific fields, e.g., earth sciences, archaeology, and forensic sciences.

A randomized trial of ibrutinib and R-GDP prior to stem cell transplant in relapsed diffuse large B-cell lymphoma.

In the LY.17 randomized phase II clinical trial, adults with relapsed and refractory diffuse large B-cell lymphoma treated with ibrutinib-R-GDP (IR-GDP) for up to three cycles had more documented bacterial and fungal infections, without improvement in overall response, compared with R-GDP. CR, complete response; DLBCL, diffuse large B-cell lymphoma; PD, progressive disease; PR, partial response; R/R, relapsed/refractory; SD, stable disease.