Therapeutic potential of siRNA PMP22-SQ nanoparticles for Charcot-Marie-Tooth 1A neuropathy in rodents and non-human primates.

Small interfering RNA (siRNA) has shown promising results for the treatment of Charcot-Marie-Tooth disease 1A (CMT1A) caused by overexpression of peripheral myelin protein (PMP22), leading to myelin dysfunction and axonal damage. Recently, we developed siRNA PMP22-squalene (SQ) nanoparticles (NPs) for intravenous use. Three consecutive injections of siRNA PMP22-SQ NPs at a cumulative dose of 1.

Prognostic value of coronary plaque composition in symptomatic patients with obstructive coronary artery disease.

Objectives: To determine whether plaque composition analysis defined by cardiac CT can provide incremental prognostic value above coronary artery disease (CAD) burden markers in symptomatic patients with obstructive CAD.

Materials And Methods: Between 2009 and 2019, a multicentric registry included all consecutive symptomatic patients with obstructive CAD (at least one ≥ 50% stenosis on CCTA) and was followed for major adverse cardiovascular (MACE) defined by cardiovascular death or nonfatal myocardial infarction. Each coronary segment was scored visually for both the degree of stenosis and composition of plaque, which were classified as non-calcified, mixed, or calcified.

Novel Real-Time Fluoroscopic Assessment Method of Transcatheter Heart Valve Expansion Following Balloon-Expandable TAVR.

Background: Transcatheter heart valve (THV) underexpansion after transcatheter aortic valve replacement may be associated with worse outcomes. THV expansion can be assessed fluoroscopically using a pigtail for calibration; however, the accuracy of this technique specific to transcatheter aortic valve replacement is unknown. We assessed the accuracy and reproducibility of a novel fluoroscopic method to assess THV expansion using the THV commissural post for calibration.

Effectiveness and safety of biosimilars in pediatric inflammatory bowel diseases: an observational longitudinal study on the French National Health Data System.

Background: Data on biosimilar use in pediatric inflammatory bowel diseases (IBD) are scarce compared to the status of studies in adults, resulting in limitations in its treatment. We compared effectiveness and safety of biosimilars versus originators in this population.

Methods: We used data from the French National Health Data System to identify children (less than 18 years old at treatment initiation) initiating treatment with a biosimilar or the originator infliximab or adalimumab for Crohn's disease (CD) or ulcerative colitis (UC), from first biosimilar launch (January 2015 and October 2018, respectively) to 31 December 2022.

Adjuvant Chemotherapy After Resection of Localized Pancreatic Adenocarcinoma Following Preoperative FOLFIRINOX.

Importance: The effect of adjuvant chemotherapy following resection of pancreatic adenocarcinoma after preoperative (m)FOLFIRINOX (combination leucovorin calcium [folinic acid], fluorouracil, irinotecan hydrochloride, and oxaliplatin in full or modified dosing) chemotherapy on overall survival (OS) is unclear because current studies do not account for the number of cycles of preoperative chemotherapy and adjuvant chemotherapy regimen.

Objective: To investigate the association of adjuvant chemotherapy following resection of pancreatic adenocarcinoma after preoperative (m)FOLFIRINOX with OS, taking into account the number of cycles of preoperative chemotherapy and adjuvant chemotherapy regimen.

Design, Setting, And Participants: This retrospective cohort study included patients with localized pancreatic adenocarcinoma treated with 2 to 11 cycles of preoperative (m)FOLFIRINOX followed by resection across 48 centers in 20 countries from 2010 to 2018.

Allergic Rhinitis and its Impact on Asthma (ARIA) Classes in MASK-air Users.

Background And Objectives: The Allergic Rhinitis and its Impact on Asthma (ARIA) guidelines classify rhinitis as "intermittent" or "persistent" and "mild" or "moderate-severe". To assess ARIA classes in a real-world study in terms of phenotypic differences and their association with asthma.

Methods: We performed a cross-sectional real-world study based on users of the MASK-air® app who reported data for at least 3 different months.

Impact of Sjögren's disease on quality of sexual life using the Qualisex score.

Objective: This study aimed to assess the impact of Sjögren disease (SjD) on the quality of sexual life and its determinants using the Qualisex questionnaire.

Methods: The Qualisex questionnaire was administered to participants within the ASSESS cohort, a French national multicentric prospective cohort of individuals with SjD. Patients' characteristics and psychometric evaluations were also collected.

Impact of frailty and older age on weaning from invasive ventilation: a secondary analysis of the WEAN SAFE study.

Objective: To understand the impact of both frailty and chronologic age on outcomes of weaning from invasive mechanical ventilation (MV).

Methods: The study population consisted of patients enrolled in the 'WorldwidE. AssessmeNt of Separation of pAtients From ventilatory assistancE (WEAN SAFE) study.

[Continuity of care and the power of narrative in perinatal psychotrauma].

With the help of clinical vignettes, the authors present their approach to maternity care for exiled pregnant women at risk of psychotrauma. They emphasize the importance of co-constructing a life story to bridge discontinuities, and of working as a team with continuity to avoid further breakdowns. At stake is the birth of a baby, which may suffer from psychotraumatic sharing without adequate support.

[The question of cultural transference and countertransference in transcultural clinical practice].

Through a clinical analysis of the cultural transfers and counter-transfers at play in the exchange between a nurse and a young unaccompanied minor, the authors propose a reflection on the question of transcultural encounters in healthcare. What recognition should be given to otherness, so as not to reduce the other to his or her difference, but rather to support him or her in the construction of an identity at the heart of the issue of métissage?

Lack of context modulation in human single neuron responses in the medial temporal lobe.

In subjects implanted with intracranial electrodes, we use two different stories involving the same person (or place) to evaluate whether and to what extent context modulates human single-neuron responses. Nearly all neurons (97% during encoding and 100% during recall) initially responding to a person/place do not modulate their response with context. Likewise, nearly none (<1%) of the initially non-responsive neurons show conjunctive coding, responding to particular persons/places in a particular context during the tasks.

Unusual Cause of Ventricular Arrhythmia.

Although relatively rare, cardiac metastases represent a significant clinical challenge because of their impact on cardiac function and overall patient prognosis. This case presents a rare and atypical presentation of a patient with ventricular arrhythmia revealing a metastatic cancer in the heart. A 59-year-old man with lung cancer was admitted for chest tightness and episodes of syncope.

Computerized cognitive training for problem gambling: A randomized controlled trial (TRAIN-online).

Background: Non-face-to-face interventions offer promise, with cognitive training showing potential but inconsistent efficacy in problem gambling.

Methods: We conducted a non-face-to-face, parallel, randomized, controlled, single-blinded trial to evaluate training programs in adults with problem gambling (CPGI ≥5). Participants were randomized 1:1 to a web-based cognitive training program targeting inhibition unrelated to addiction cues or a control program on visuo-spatial functioning.

A Machine Learning Model Using Cardiac CT and MRI Data Predicts Cardiovascular Events in Obstructive Coronary Artery Disease.

Background Multimodality imaging is essential for personalized prognostic stratification in suspected coronary artery disease (CAD). Machine learning (ML) methods can help address this complexity by incorporating a broader spectrum of variables. Purpose To investigate the performance of an ML model that uses both stress cardiac MRI and coronary CT angiography (CCTA) data to predict major adverse cardiovascular events (MACE) in patients with newly diagnosed CAD.

Girolline is a sequence context-selective modulator of eIF5A activity.

Natural products have a long history of providing probes into protein biosynthesis, with many of these compounds serving as therapeutics. The marine natural product girolline has been described as an inhibitor of protein synthesis. Its precise mechanism of action, however, has remained unknown.

Clinical Outcomes According to the Extent of Atherosclerotic Disease in Female Patients Undergoing Transcatheter Aortic Valve Replacement: An Analysis From the WIN-TAVI Registry.

Background: Limited data exist on the impact of polyvascular disease (PolyVD) on clinical outcomes in female patients undergoing transcatheter aortic valve replacement (TAVR). We therefore sought to investigate clinical outcomes in women with versus without PolyVD undergoing TAVR.

Methods: Female participants from the multicentre Women's International Transcatheter Aortic Valve Implantation (WIN-TAVI) registry were categorized based on the presence or absence of PolyVD.

High-risk percutaneous coronary intervention in patients with reduced left ventricular ejection fraction deemed not suitable for surgical revascularisation. A clinical consensus statement from the European Association of Percutaneous Cardiovascular Interventions (EAPCI) in collaboration with the ESC Working Group on Cardiovascular Surgery.

This clinical consensus statement of the European Association of Percutaneous Cardiovascular Interventions was developed in association with the European Society of Cardiology Working Group on Cardiovascular Surgery. It aims to define procedural and contemporary technical requirements that may improve the efficacy and safety of percutaneous coronary intervention (PCI), both in the acute phase and at long-term follow-up, in a high-risk cohort of patients on optimal medical therapy when clinical and anatomical high-risk criteria are present that entail unacceptable surgical risks, precluding the feasibility of coronary artery bypass grafting (CABG). This document pertains to patients with surgical contraindication according to the Heart Team, in whom medical therapy has failed (e.

Prognostic Value of Cardiac MRI Late Gadolinium Enhancement Granularity in Participants with Ischemic Cardiomyopathy.

Background Ischemic late gadolinium enhancement (LGE) assessed with cardiac MRI is a well-established prognosticator in ischemic cardiomyopathy. However, the prognostic value of additional LGE parameters, such as extent, transmurality, location, and associated midwall LGE, remains unclear. Purpose To assess the prognostic value of ischemic LGE features to predict all-cause mortality in ischemic cardiomyopathy.

[A national survey from the French Society of Pathology on research and application of artificial intelligence in pathology].

The 2030 French Innovation Healthcare national plan defined "Digital Healthcare" as one of its priority. The deployment of digital pathology and artificial intelligence fits perfectly into this framework. Therefore we evaluated the pathologist's interest in those fields by conducting an online survey among members of the French Society of Pathology, mainly composed of senior pathologists and trainees (n=2301).

Does Asthma Affect the Risk of Developing Breast Cancer?

Background: The role of the immune system in cancer defense is likely underappreciated. While there has been longstanding interest in the role of atopic diseases in cancer, only a few studies have tested this hypothesis.

Methods: We analyzed data from 202,055 women participating in the Nurses' Health Study (NHS) and the Nurses' Health Study II (NHS II) to explore whether asthma is associated with breast cancer.

Nanopore Sequencing as a Cutting-Edge Technology for Medulloblastoma Classification.

Background: Medulloblastoma (MB) is one of the most prevalent embryonal malignant brain tumors. Current classification organizes these tumors into four molecular subgroups (WNT, SHH, Group 3, and Group 4 MB). Recently, a comprehensive classification has been established, identifying numerous subtypes, some of which exhibit a poor prognosis.

Stable Convergent Polyneuronal Innervation and Altered Synapse Elimination in Muscles from Patients with Blepharospasm Responding Poorly to Recurrent Botulinum Type-A Neurotoxin Injections.

Botulinum neurotoxin type-A (BoNT/A), which blocks quantal acetylcholine (ACh) release at the neuromuscular junction (NMJ), has demonstrated its efficacy in the symptomatic treatment of blepharospasm. In 3.89% of patients treated for blepharospasm at Tenon Hospital, BoNT/A was no longer effective in relieving the patient's symptoms, and a partial upper myectomy of the muscle was performed.