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Surgical treatment of congenital pseudarthrosis of the Tibia in children: CPAM-LRC consensus and guidelines.
Int J Surg
January 2025
Department of Orthopedics, Shanghai Sixth People's Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Purpose: Congenital pseudarthrosis of the tibia (CPT) is a rare condition typically manifesting within the first decade of life. The primary objectives of surgical intervention for CPT include achieving long-term bony union of the tibia, preventing or minimizing limb length discrepancies (LLD), avoiding mechanical axis deviations of the tibia and adjacent joints, and preventing refracture. This study aims to conduct a systematic review of current treatment methods for CPT to determine the most effective non-surgical and surgical management strategies for pediatric patients with this condition.
View Article and Find Full Text PDFDifferent Times for Different Metrics: Predicting 90 Days of Intermittently Scanned Continuous Glucose Monitoring Data in Subjects With Type 1 Diabetes on Multiple Daily Injection Therapy. Findings From a Multicentric Real-World Study.
J Diabetes Sci Technol
January 2025
Unit of Endocrine Diseases and Diabetology, Department of Medicine, ASST Papa Giovanni XXIII, Bergamo, Italy.
Aims: According to the 2023 International Consensus, glucose metrics derived from two-week-long continuous glucose monitoring (CGM) can be extrapolated up to 90 days before. However, no studies have focused on adults with type 1 diabetes (T1D) on multiple daily injections (MDIs) and with second-generation intermittently scanned CGM (isCGM) sensors in a real-world setting.
Methods: This real-world, retrospective study included 539 90-day isCGM data from 367 adults with T1D on MDI therapy.
Medication non-adherence: reflecting on two decades since WHO adherence report and setting goals for the next twenty years.
Front Pharmacol
December 2024
Syreon Research Institute, Budapest, Hungary.
Background: Non-adherence to medication remains a persistent and significant challenge, with profound implications for patient outcomes and the long-term sustainability of healthcare systems. Two decades ago, the World Health Organization (WHO) dedicated its seminal report to adherence to long-term therapies, catalysing notable changes that advanced both research and practice in medication adherence. The aim of this paper was to identify the most important progress made over the last 2 decades in medication adherence management and to initiate a discussion on future objectives, suggesting priority targets for the next 20 years.
View Article and Find Full Text PDFCutoffs on severity metrics for minimal manifestations or better status in patients with generalized myasthenia gravis.
Front Immunol
December 2024
Department of Neurology, Hanamaki General Hospital, Hanamaki, Japan.
International consensus guidance and Japanese clinical guidelines for myasthenia gravis (MG) recommend achieving minimal manifestations or better status (MM-or-better) as the severity component of the treatment goal. However, the subjective nature of determining MM can result in ambiguity regarding this category in clinical practice and clinical trials. This study analyzed severity metrics in a large number of MG patients to propose criteria for MM-or-better.
View Article and Find Full Text PDFLong-term neuropsychologic outcome of pre-emptive mTOR inhibitor treatment in children with tuberous sclerosis complex (TSC) under 4 months of age (PROTECT), a two-arm, randomized, observer-blind, controlled phase IIb national multicentre clinical trial: study protocol.
Orphanet J Rare Dis
January 2025
Division of Pediatric Epileptology, Department of Pediatrics I, Medical Faculty of Heidelberg, Heidelberg University, Heidelberg, Germany.
Background: Tuberous sclerosis complex (TSC) is an autosomal dominant genetic disorder affecting multiple organ systems, with a prevalence of 1:6,760-1:13,520 live births in Germany. On the molecular level, TSC is caused by heterozygous loss-of-function variants in either of the genes TSC1 or TSC2, encoding the Tuberin-Hamartin complex, which acts as a critical upstream suppressor of the mammalian target of rapamycin (mTOR), a key signaling pathway controlling cellular growth and metabolism. Despite the therapeutic success of mTOR inhibition in treating TSC-associated manifestations, studies with mTOR inhibitors in children with TSC above two years of age have failed to demonstrate beneficial effects on disease-related neuropsychological deficits.
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