Background: As people with cystic fibrosis (PWCF) live longer due to the breakthrough drug elexacaftor-tezacaftor-ivacaftor (ETI), they have questioned whether other CF therapies could be safely discontinued. SIMPLIFY was the first prospective, randomized trial to evaluate non-inferiority of discontinuing versus continuing two therapies. The QUEST (Qualitative Understanding of Experiences in the SIMPLIFY Trial) study was conducted to understand experiences of PWCF enrolled in SIMPLIFY, including why they joined, perceptions of randomization, decision-making around study withdrawal, and considerations for future discontinuation studies.
View Article and Find Full Text PDFT-cell acute lymphoblastic leukemia (T-ALL) is an aggressive hematological malignancy that expresses high levels of the enzyme aldo-keto reductase family 1 member C3 (AKR1C3). To exploit this finding, we developed a novel prodrug, ACHM-025, which is selectively activated by AKR1C3 to a nitrogen mustard DNA alkylating agent. We show that ACHM-025 has potent in vivo efficacy against T-ALL patient-derived xenografts (PDXs) and eradicated the disease in 7 PDXs.
View Article and Find Full Text PDFBackground: Since 2013, the Veterans Health Administration (VHA) has advanced a person-centered, Whole Health (WH) System of Care, a shift from a disease-oriented system to one that prioritizes "what matters most" to patients in their lives. Whole Health is predicated on patient-provider interactions marked by a multi-level understanding of health and trusted relationships that promote well-being. Presently, WH implementation has been focused largely in primary care settings, yet the goal is to effect a system-wide transformation of care so that Veterans receive WH across VHA clinical settings, including specialty care.
View Article and Find Full Text PDFThe laboratory mouse has been described as a "miracle" model organism, providing a window by which we may gain an understanding of ourselves. Since the first recorded mouse experiment in 1664, the mouse has become the most used animal model in biomedical research. Mice are ideally suited as a model organism because of their small size, short gestation period, large litter size, and genetic similarity to humans.
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