Cemdomespib Therapy Slows the Progression of Neuromuscular Weakness and Demyelination in the R75W-Connexin 32 Animal Model of Charcot-Marie-Tooth 1X Disease.

Mutations in connexin 32 (Cx32) are a common cause of Charcot-Marie-Tooth 1X (CMT1X) disease, an inherited peripheral neuropathy characterized by progressive neuromuscular weakness and demyelination. There are no approved pharmacologic therapies for CMT1X, and identifying new treatments that slow the onset and severity of neuromuscular decline may aid disease management. Cemdomespib is an orally bioavailable small molecule that improved demyelination and neuromuscular junction (NMJ) morphology in mice lacking Cx32 expression.

Living with Pediatric Coeliac Disease: Lessons for Health Service Delivery.

Background: Coeliac Disease (CD) affects up to 1.4% of children worldwide, with a rising global incidence. A less typical clinical presentation and the need for a life-long gluten exclusion diet raise challenges for diagnosis, management, and healthcare delivery with considerable impacts for CD patients and families as well as clinical services.

Secretory breast carcinoma: a multicenter clinicopathologic study of 80 cases with emphasis on prognostic analysis and chemotherapy benefit.

Purpose: To investigate clinicopathologic characteristics and prognosis in secretory breast carcinoma (SBC) and to determine chemotherapy benefits stratified by different subgroups.

Methods: SBCs and triple-negative invasive ductal carcinoma patients (TN-IDCs) were enrolled from three cancer centers between January 2011 and December 2020. SBCs were further divided into two subgroups: those with triple negativity (TN-SBCs) and those without (non-TN-SBCs).

Chinese expert consensus on sequential surgery following conversion therapy based on combination of immune checkpoint inhibitors and antiangiogenic targeted drugs for advanced hepatocellular carcinoma (2024 edition).

Up to half of hepatocellular carcinoma (HCC) cases are diagnosed at an advanced stage, for which effective treatment options are lacking, resulting in a poor prognosis. Over the past few years, the combination of immune checkpoint inhibitors and anti-angiogenic targeted therapy has proven highly efficacious in treating advanced HCC, significantly extending patients' survival and providing a potential for sequential curative surgery. After sequential curative hepatectomy or liver transplantation following conversion therapy, patients can receive long-term survival benefits.

Brief Report: Protease Inhibitors Versus Nonnucleoside Reverse Transcriptase Inhibitors and the Risk of Cancer Among People With HIV.

Background: The effect of initial antiretroviral therapy (ART) class on cancer risk in people with HIV (PWH) remains unclear.

Setting: Cohort study of 36,322 PWH enrolled (1996-2014) in the North American AIDS Cohort Collaboration on Research and Design.

Methods: We followed individuals from ART initiation (protease inhibitor [PI]-, non-nucleoside reverse transcriptase inhibitor [NNRTI]-, or integrase strand transfer inhibitor [INSTI]-based) until incident cancer, death, loss-to-follow-up, 12/31/2014, 85 months (intention-to-treat analyses [ITT]), or 30 months (per-protocol [PP] analyses).