Myositis/myasthenia gravis caused by immune checkpoint inhibition: A report of two cases and a brief review of the literature.

Immune checkpoint inhibitors (ICIs) have revolutionized cancer therapy by enhancing the ability of the immune system to combat malignancies. Nivolumab and cemiplimab, monoclonal antibodies targeting programmed cell death protein 1, have exhibited notable therapeutic efficacy; however, they are associated with immune-related adverse events (irAEs). The present study describes the cases of 2 patients, a 71-year-old male with metastatic esophageal adenocarcinoma and a 66-year-old female with metastatic squamous cell carcinoma who developed acute/subacute onset rapidly progressive myositis/myasthenia gravis (MG) following treatment with nivolumab and cemiplimab.

Reversion of metabolic dysfunction-associated steatohepatitis by skeletal muscle-directed FGF21 gene therapy.

The highly prevalent metabolic dysfunction-associated steatohepatitis (MASH) is associated with liver steatosis, inflammation, and hepatocyte injury, which can lead to fibrosis and may progress to hepatocellular carcinoma and death. New treatment modalities such as gene therapy may be transformative for MASH patients. Here, we describe that one-time intramuscular administration of adeno-associated viral vectors of serotype 1 (AAV1) encoding native fibroblast growth factor 21 (FGF21), a key metabolic regulator, resulted in sustained increased circulating levels of the factor, which mediated long-term (>1 year) MASH and hepatic fibrosis reversion and halted development of liver tumors in obese male and female mouse models.

Understanding Community-Specific Health Behaviors to Prevent Type 2 Diabetes and Depression in Rural Adolescents: A Qualitative Study.

Background: Type 2 diabetes (T2D) and depression co-occur, and rates are on the rise in adolescents, disproportionately affecting teenagers in rural communities and those who identify as members of historically disadvantaged racial/ethnic groups. Addressing the promotion of health behaviors is important for prevention of these comorbid health concerns; however, disparities in their prevalence highlight that a healthy lifestyle is not equally accessible for all individuals. Thus, holistic and multi-level approaches that address structural inequities, leverage cultural and family assets, and are effectively integrated into the community are critically needed.

Value contribution of etranacogene dezaparvovec gene therapy in moderately severe and severe haemophilia B through multi-criteria decision analysis.

Introduction: The value of gene therapies for haemophilia needs to be assessed holistically.

Aim: To determine the value of etranacogene dezaparvovec (ED) compared to current extended half-life (EHL) recombinant factors (rFIX), using multi-criteria decision analysis (MCDA).

Method: MCDA EVIDEM methodology adapted to orphan drugs was used, with nine quantitative criteria and four contextual criteria.

CD11b suppresses TLR7-driven inflammatory signaling to protect against lupus nephritis.

Lupus Nephritis (LN) is a severe complication of systemic lupus erythematosus (SLE) that affects kidney function. Here, we investigated the role of CD11b, a protein encoded by the gene, in the development of LN and its functional activation as a therapeutic strategy. Genetic coding variants of significantly increase the risk for SLE and LN by producing a less active CD11b and leading to elevated levels of type I interferon (IFN I).