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Roadmap to advance therapeutics for -related disorder: a patient organization perspective from SynGAP Research Fund.
Ther Adv Rare Dis
January 2025
SynGAP Research Fund, 2856 Curie Pl., San Diego, CA 92122, USA.
-related disorder (SRD) is a developmental and epileptic encephalopathy caused by a disruption of the gene. At the beginning of 2024, it is one of many rare monogenic brain disorders without disease-modifying treatments, but that is changing. This article chronicles the last 5 years, beginning when treatments for SRD were not publicly in development, to the start of 2024 when many SRD-specific treatments are advancing.
View Article and Find Full Text PDFCore Concepts: Self-Controlled Designs in Pharmacoepidemiology.
Pharmacoepidemiol Drug Saf
January 2025
Department of Non-communicable Disease Epidemiology, London School of Hygiene and Tropical Medicine, London, UK.
One of the key challenges in pharmacoepidemiological studies is that of uncontrolled confounding, which occurs when confounders are poorly measured, unmeasured or unknown. Self-controlled designs can help address this issue, as their key comparison is not between people, but periods of time within the same person. This controls for all time-stable confounders (genetics) and in the absence of time-varying confounding negates the need for an external control group.
View Article and Find Full Text PDFPoint of view: Challenges in implementation of new immunotherapies for Alzheimer's disease.
J Prev Alzheimers Dis
January 2025
Division of Neurogeriatrics, Department of Neurobiology, Care Sciences and Society, Karolinska Institutet, BioClinicum, 171 64 Solna, Sweden; Theme Inflammation and Aging, Karolinska University Hospital, 141 86 Stockholm, Sweden.
The advancement of disease-modifying treatments (DMTs) for Alzheimer's disease (AD), along with the approval of three amyloid-targeting therapies in the US and several other countries, represents a significant development in the treatment landscape, offering new hope for addressing this once untreatable chronic progressive disease. However, significant challenges persist that could impede the successful integration of this class of drugs into clinical practice. These challenges include determining patient eligibility, appropriate use of diagnostic tools and genetic testing in patient care pathways, effective detection and monitoring of side effects, and improving the healthcare system's readiness by engaging both primary care and dementia specialists.
View Article and Find Full Text PDFFirst-in-human phase I trial of the bispecific CD47 inhibitor and CD40 agonist Fc-fusion protein, SL-172154 in patients with platinum-resistant ovarian cancer.
J Immunother Cancer
January 2025
Medical Oncology, Sarah Cannon Research Institute, Nashville, Tennessee, USA.
Background: SL-172154 is a hexameric fusion protein adjoining the extracellular domain of SIRPα to the extracellular domain of CD40L via an inert IgG-derived Fc domain. In preclinical studies, a murine equivalent SIRPα-Fc-CD40L fusion protein provided superior antitumor immunity in comparison to CD47- and CD40-targeted antibodies. A first-in-human phase I trial of SL-172154 was conducted in patients with platinum-resistant ovarian cancer.
View Article and Find Full Text PDFThe IL-23R and Its Genetic Variants: A Hitherto Unforeseen Bridge Between the Immune System and Cancer Development.
Cancers (Basel)
December 2024
Immuno-Genetics Lab, Department of Health Science, Medical School, University "Magna Graecia" of Catanzaro, 88100 Catanzaro, Italy.
IL-23R (interleukin-23 receptor), found on the surface of several immune cells, plays a key role in the immune system. Indeed, this process is not limited to the inflammatory response but also plays a role in the adaptive immune response. The binding between IL-23R and its specific ligand, the interleukin 23, initiates a number of specific signals by modulating both properties and behavior of immune cells.
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