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Preclinical evaluation of the efficacy and safety of AAV8-TNAP-D10 in Alpl-/- and AlplPrx1/Prx1 mouse models for the treatment of early and late-onset hypophosphatasia.
J Bone Miner Res
January 2025
Human Genetics Program, Sanford Burnham Prebys Medical Discovery Institute, La Jolla, CA, United States.
We previously documented successful resolution of skeletal and dental disease in the infantile and late-onset murine models of hypophosphatasia (HPP), with a single injection of an adeno-associated serotype 8 vector encoding mineral-targeted TNAP (AAV8-TNAP-D10). Here, we conducted dosing studies in both HPP mouse models. A single escalating dose from 4x108 up to 4x1010 (vg/b) was intramuscularly injected into 4-day-old Alpl-/- mice (an infantile HPP model) and a single dose from 4x106 up to 4x109 (vg/b) was administered to 8-week-old AlplPrx1/Prx1 mice (a late-onset HPP model).
View Article and Find Full Text PDFMethodological standards for comparative effectiveness research: considerations for evidentiary review for medicare drug price negotiation.
Expert Rev Pharmacoecon Outcomes Res
January 2025
IQVIA, Durham, NC.
Introduction: The 2022 Inflation Reduction Act (IRA) is expected to result in lower drug prices for Medicare beneficiaries in the United States (US). The Centers for Medicare & Medicaid Services (CMS) released the most recent draft guidance for the Medicare Drug Price Negotiation (DPN) program in May 2024.
Areas Covered: In August 2023, the list of 10 drugs selected for the DPN were published and the first round of negotiations are now complete.
Prevalence of chronic kidney disease and anemia in Hirakud Command Area, Odisha, India: unveiling the role of environmental toxicants.
J Nephrol
January 2025
Laboratory of Renal Toxicopathology & Medicine, P.G. Department of Environmental Sciences, Sambalpur University, Burla, Odisha, 768019, India.
Background: The present community-based study assessed the prevalence of chronic kidney disease (CKD)/chronic kidney disease of unknown origin (CKDu) as well as anemia in some intense agricultural zones under Hirakud Command Area and evaluated their association with pesticides and heavy metal exposure.
Methods: Random cluster sampling method was used to assess the prevalence of CKD and anemia. Hematological analysis was carried out using autoanalyzer.
Purpose: Heart failure (HF) is a disease that leads to approximately 300,000 fatalities annually in Europe and 250,000 deaths each year in the United States. Type 2 Diabetes Mellitus (T2DM) is a significant risk factor for HF, and testing for N-terminal (NT)-pro hormone BNP (NT-proBNP) can aid in early detection of HF in T2DM patients. We therefore developed and validated the HFriskT2DM-HScore, an algorithm to predict the risk of HF in T2DM patients, so guiding NT-proBNP investigation in a primary care setting.
View Article and Find Full Text PDFAssociation between extracellular matrix protein 1 (ECM1) gene polymorphisms (rs3834087 and rs3754217) and Hepatitis B Virus evolution in an African cohort.
Cell Mol Biol (Noisy-le-grand)
January 2025
Université Joseph KI-ZERBO, Laboratoire de Biologie Moléculaire et de Génétique (LABIOGENE), 03 BP 7021 Ouagadougou 03, Burkina Faso.
Article Synopsis
- Hepatitis B virus (HBV) is a major global health concern linked to liver disease and cancer, with research focusing on genetic factors that affect its evolution.
- Recent studies highlighted the ECM1 gene, specifically two polymorphisms (rs3834087 and rs3754217), which may influence HBV pathogenesis, particularly in an African cohort analyzed in this research.
- The study found that the heterozygous genotype of rs3754217 appears to protect against chronic hepatitis, suggesting that certain genetic variations may impact the severity of the disease in infected individuals.
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