Risultati per: Springer: Nuove riviste open access di Medicina Generale

New England Journal of Medicine, Ahead of Print.

Our results suggest that losartan was well tolerated by children with RDEB, and provide preliminary evidence that it may reduce disease burden. Further research with larger sample sizes and longer durations is needed to establish the treatment’s long-term efficacy and safety.

Introduction
The diagnosis of asthma is often based on characteristic patterns of symptoms in the absence of an alternative explanation, resulting in over and under diagnosis. Therefore, diagnostic guidelines usually recommend including confirmation of variable airflow obstruction. Some recommend using a sequence of objective tests; however the tests used, the specific cut-off values and the specified order are yet to be validated. We aimed to determine the optimal cut-off values and series of investigations to diagnose asthma. We also explore the potential for novel tests of small airways function and biomarkers, which could be incorporated into future diagnostic pathways.

Methods and analysis
The Rapid Access Diagnostics for Asthma study is an observational study of 300 symptomatic patients with ‘clinician-suspected asthma’ and healthy controls (aged ≥3 to

A Milano la Convention Anmco per ottimizzare le terapie

Stroke, Ahead of Print. BACKGROUND:The availability of mechanical thrombectomy (MT) for acute ischemic stroke is limited, and vast disparities exist between countries. We aim to create a MT access score to measure the drivers of access to help quantify and accelerate treatment worldwide.METHODS:We used a systematic review complemented by a modified Delphi method. In the first of 3 rounds, 4 independent investigators performed a systematic literature review using key search terms that drive MT access, following Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines. In the second round, a panel of 6 anonymous international experts selected key attributes needed for scoring. In the final round, a total of 12 attributes were selected on consensus, each given a score on a 0 to 3 scale. An ultimate MT access score (range, 0–36) was proposed as a new tool to use in identifying barriers to MT access and assist in providing an initial framework for public health interventions.RESULTS:Of 2864 abstracts screened, 121 studies were included in the final systematic review. A total of 34 attributes that potentially drive MT access were initially identified. In the final round, 12 attributes were selected by the expert panel: public awareness, emergency medical services transportation, prehospital large vessel occlusion screening, interhospital transfer policy, emergency department protocols, stroke imaging protocols, emergency department stroke expertise or telestroke availability, interventionalists, MT-capable centers, device availability, and insurance coverage. These attributes were weighted as part of the final score of 0 to 36.CONCLUSIONS:The MT access score represents the first tool to quantify barriers to global MT access. Its implementation stands not just as an academic achievement but as a beacon of hope for improving stroke care and outcomes worldwide, bringing us a step closer to bridging the gap in stroke treatment disparities.

The primary endpoint of time to sustained clinical improvement for distinct asunercept arms compared to SOC failed to meet statistical significance. The compound was safe and well tolerated.

Platea di 1112 ragazzi di 13 scuole. ‘Insegnare valore ricerca’

Annals of Internal Medicine, Ahead of Print.

Introduction
Lafora disease (LD) is an ultrarare fatal progressive myoclonic epilepsy, causing drug-resistant epilepsy, myoclonus and psychomotor deterioration. LD is caused by mutations in EPM2A or NHLRC1, which lead to the accumulation of polyglucosans in the brain and neurodegeneration. There are no approved treatments for LD. VAL-1221 is a fusion protein comprising the Fab portion of a cell-penetrating antibody and recombinant human acid alpha glucosidase, and has demonstrated an ability to clear polyglucosans. We hypothesise that intravenous infusion of VAL-1221 might be able to degrade cerebral polyglucosans and stabilise or improve disease outcomes. The aim of this study is to assess the safety and preliminary efficacy of VAL-1221 in patients with LD.

Methods and analysis
The study is a phase 2, single-arm, open-label, baseline-controlled clinical trial which will be conducted in a single investigational study centre in Italy, namely the sponsor ‘IRCCS Istituto delle Scienze Neurologiche di Bologna—Azienda USL di Bologna’. The study will enrol six genetically confirmed patients with mid- to late-stage LD. The global duration of the study for each participant will be 18 months, including screening period, open-label treatment (12 months) and follow-up period. VAL-1221 20 mg/kg will be administered as an intravenous infusion every week for 3 weeks, then every other week. Patients will undergo full clinical assessments at baseline, at an intermediate and at the end-of-treatment visit. The primary objective is to evaluate the safety. The exploratory efficacy endpoints will be related to epilepsy, neuropsychological and motor functions, global assessment and disease burden, in addition to biomarkers. Statistical analyses will be primarily descriptive.

Ethics and dissemination
The study protocol was approved by the local ethics committee (number 232-2023-FARM-AUSLBO-23020, 22 March 2023). The results of this study will be disseminated by the investigators through presentations at international scientific conferences and reported in peer-reviewed scientific journals.

Trial registration number
European Union Clinical Trials Register (EudraCT 2023-000185-34).

‘Positivo 30 mila nuove assunzioni tra medici e infermieri’

Ma graduatoria dopo il primo semestre sulla base dell’esito degli esami

Contenuti con creator e guide per migliorare il benessere online

Introduction
The role of sodium-glucose cotransporter 2 inhibitors (SGLT2is) in diabetes treatment is expanding; however, few studies have investigated the efficacy and safety of combining SGLT2is with insulin pump therapy. Notably, there is a scarcity of high-quality, multicentre, clinical trials. Therefore, we aim to conduct a prospective multicentre, randomised, controlled, study to investigate whether treatment of type 2 diabetes patients with continuous subcutaneous insulin infusion (CSII) combined with henagliflozin can reduce the time required for blood glucose control, decrease total insulin requirements, mitigate blood glucose fluctuations and enhance beta-cell function.

Methods and analysis
In this inpatient, open-label, multicentre, randomised, controlled trial, 200 patients with type 2 diabetes who have not received hypoglycaemic drugs will be randomly allocated at a 1:1 ratio to either the henagliflozin combined with CSII group or the CSII group. The efficacy and safety of treatment in both groups will be compared. We will use a real-time continuous glucose monitoring system for blood glucose monitoring. The primary aim of this study is to compare the time (% time in range (TIR)) in the range of 3.9~10.0 mmol/L blood glucose between the two treatment groups. The secondary outcome measures will include comparisons of the two treatment groups with respect to the (a) time at TIR >70%; (b) mean amplitude of glycaemic excursions; (c) time below range; (d) total insulin dosage; and (e) time above range.

Ethics and dissemination
This study was approved by the Ethics Committee of the First Affiliated Hospital of Guangxi Medical University and is to be conducted in accordance with the Declaration of Helsinki and Good Clinical Practice. The study will be disseminated through peer-reviewed publications and conference presentations.

Trial registration number
NCT05677334.

Nella città campana il 49esimo Congresso della Società di Storia internazionale della Medicina