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| http://dx.doi.org/10.1016/j.jdcr.2024.08.021 | DOI Listing |
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Drug Development.
Alzheimers Dement
December 2024
GSK R&D, Stevenage, Hertfordshire, United Kingdom.
Background: Genetic variants in GRN, the gene encoding progranulin, are causal for or are associated with the risk of multiple neurodegenerative diseases. Modulating progranulin has been considered as a therapeutic strategy for neurodegenerative diseases including Frontotemporal Dementia (FTD) and Alzheimer's Disease (AD). Here, we integrated genetics with proteomic data to determine the causal human evidence for the therapeutic benefit of modulating progranulin in AD.
View Article and Find Full Text PDFDrug Development.
Alzheimers Dement
December 2024
Department of Biomedical Engineering, McGill University, Montreal, QC, Canada.
Background: Randomized placebo-controlled trials (RCTs) are the gold standard to evaluate efficacy of new drug treatments for Alzheimer's disease. For example, the United States FDA approved the brain amyloid-targeting drug lecanemab following CLARITY AD, Biogen and Eisai's Phase 3 RCT. However, recruiting enough participants for a high-powered and demographically representative trial is difficult and expensive.
View Article and Find Full Text PDFBackground: Homozygosity for the rare APOE3-Christchurch (APOE3Ch) variant, encoding for apoE3-R136S (apoE3-Ch), was linked to resistance against an aggressive form of familial Alzheimer's disease (AD). Carrying two copies of APOE3Ch was sufficient to delay autosomal AD onset by 30 years. This remarkable protective effect makes it a strong candidate for uncovering new therapies against AD.
View Article and Find Full Text PDFOutcomes of Immunotherapy Treatment in Sinonasal Mucosal Melanoma.
Am J Rhinol Allergy
January 2025
Department of Otorhinolaryngology-Head and Neck Surgery, University of Pennsylvania, Perelman School of Medicine, Philadelphia, Pennsylvania.
Background: Sinonasal mucosal melanoma has poor survival despite multimodality treatment. While the impact of immunotherapy (IT) on metastatic cutaneous melanoma is well-defined, there are relatively little data on sinonasal mucosal melanoma.
Objective: We sought to define immunotherapy outcomes in patients with sinonasal mucosal melanoma.
Dementia Care Research and Psychosocial Factors.
Alzheimers Dement
December 2024
Department of Clinical, Educational, and Health Psychology, Division of Psychology and Language Sciences, University College London, London, United Kingdom.
Background: How people affected by non-memory-led and inherited dementias (NMLDs) interact with online health resources is poorly understood. We conducted the world's largest survey exploring 'digital access in non-memory-led dementias' to learn directly from people with NMLD, their care partners and NMLD Healthcare Professionals (HCPs) about the NMLD experience interacting with web-based health resources.
Method: Four surveys [for individuals diagnosed with NMLD, care partners, care partner proxy for person with NMLD, HCP proxy] were co-developed with people with NMLD experience.
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