Meta-analysis of the efficacy and safety of vamorolone in Duchenne muscular dystrophy.

Background: Duchenne muscular dystrophy (DMD) is a severe neuromuscular disorder, often leading to wheelchair dependence by age 13 with limited treatment options, largely relying on glucocorticosteroids. We assessed the efficacy and safety of vamorolone, a modified synthetic corticosteroid, for DMD.

Methods: We performed a systematic review and meta-analysis using seven databases including prospective studies comparing vamorolone with glucocorticosteroids or placebo in DMD patients.

Prognostic value of metabolic tumor volume on [F]FDG PET/CT in addition to the TNM classification system of locally advanced non-small cell lung cancer.

Purpose: Staging of non-small cell lung cancer (NSCLC) is commonly based on [F]FDG PET/CT, in particular to exclude distant metastases and guide local therapy approaches like resection and radiotherapy. Although it is hoped that PET/CT will increase the value of primary staging compared to conventional imaging, it is generally limited to the characterization of TNM. The first aim of this study was to evaluate the PET parameter metabolic tumor volume (MTV) above liver background uptake as a prognostic marker in lung cancer.

Single-Cell RNA Sequencing to Guide Autologous Preterm Cord Mesenchymal Stromal Cell-Therapy.

Rationale: The chronic lung disease bronchopulmonary dysplasia (BPD) remains the most common complication of extreme prematurity (<28 weeks of gestation). Umbilical cord-derived mesenchymal stromal cells (UC-MSCs) represent an opportunity for autologous cell-therapy, as UC-MSCs have been shown to improve lung function and structure in experimental BPD. However, characterization and repair capacity of UC-MSCs derived from donors with pregnancy-related complications associated with prematurity remain unexplored.

Depletion of macrophages and osteoclast precursors mitigates iron overload-mediated bone loss.

Iron is an essential element for physiological cellular processes, but is toxic in excess. Iron overload diseases are commonly associated with low bone mass. Increased bone resorption by osteoclasts as well as decreased bone formation by osteoblasts have been implicated in bone loss under iron overload conditions.

MS brain health quality standards: a survey on the reality in clinical practice in Germany.

Background: The quality of treatment is especially critical in the context of complex and chronic diseases such as multiple sclerosis (MS). The Brain Health Initiative, an independent international consortium of neurologists, reached a consensus on time-based quality standards prioritizing brain health-focused care for people with MS.

Objectives: To gain deeper insights into the transferability of these quality standards to a specific area, we conducted a survey among MS experts across various MS centers in Germany.

Bone phenotyping of murine hemochromatosis models with deficiencies of Hjv, Alk2, or Alk3: The influence of sex and the bone compartment.

Osteopenia is frequently observed in patients with iron overload, especially in those with HFE-dependent hereditary hemochromatosis (HH). Interestingly, not all mouse models of HH show bone loss, suggesting that iron overload alone may not suffice to induce bone loss. In this study, the bone phenotypes of Hjv and hepatocyte-specific Alk2- and Alk3-deficient mice as additional mouse models of HH were investigated to further clarify, how high iron levels lead to bone loss and which signaling mechanisms are operational.

Validation of the Revised 2022 European LeukemiaNet Risk Stratification in Adult Patients with Acute Myeloid Leukemia.

In 2022, the European LeukemiaNet (ELN) risk stratification for patients with AML has been updated. We aimed to validate the prognostic value of the 2022 ELN classification (ELN22) evaluating 1,570 newly diagnosed AML patients (median age, 56 years) treated with cytarabine-based intensive chemotherapy regimens. As compared to the 2017 ELN classification (ELN17), allocating 595 (38%), 413 (26%) and 562 (36%) patients to the favorable, intermediate and adverse risk category, ELN22 risk was favorable, intermediate, and adverse in 575 (37%), 410 (26%), and 585 (37%) patients, respectively.

'You Also Have to Let People Go'-Effects of Formative Experiences with Dying and Death on Medical Trainees' Attitudes.

This study investigates the effects of medical students' and residents' formative patient death experiences on their understanding of the role of the physician in dealing with dying patients. Analyses revealed a change in attitude, an acceptance of death as 'part of life'. Thoughtful and comprehensive care, allowing patients to die and enabling them to have a beautiful death, were identified as the physician's duty.

Do We Need More Structured MD Thesis Programs? A Propensity Score Matched Analysis of the Research Program at the Medical Faculty Dresden.

Introduction: Conducting a Medical Doctorate (MD) thesis is desired by the majority of medical students. However, the needed scientific competencies are not regularly implemented in medical education. To support students during their MD thesis, a graduate college was implemented.

Heart rate variability biofeedback for critical illness polyneuropathy: a randomized sham-controlled study.

Background And Purpose: Critical illness polyneuropathy (CIP) has been linked to neurocardiac dysfunction mediated by autonomic nervous system dysregulation, which increases mortality. We aimed to assess if heart rate variability (HRV) biofeedback could improve neurocardiac function in CIP.

Methods: We randomly allocated (1:1) patients with electrophysiologically confirmed CIP undergoing early inpatient neurological rehabilitation to additional HRV or sham biofeedback over 14 days.

VISIT STROKE: non-inferiority of telemedicine-based neurological consultation for post-acute stroke patients - protocol of a prospective observational controlled multi-center study.

Background: Telemedicine provides specialized medical expertise in underserved areas where neurological expertise is frequently not available on a daily basis for hospitalized stroke patients. While tele-consultations are well established in acute stroke assessment, the value of telemedicine-based ward-rounds in the subsequent in-patient stroke management is unknown.

Methods: Four telemedicine stroke networks in Germany, implemented in eight out of 16 federal states, participate in this prospective observational multi-center study.

Endovascular thrombectomy for childhood stroke (Save ChildS Pro): an international, multicentre, prospective registry study.

Background: Emerging evidence suggests that endovascular thrombectomy is beneficial for treatment of childhood stroke, but the safety and effectiveness of endovascular thrombectomy has not been compared with best medical treatment. We aimed to prospectively analyse functional outcomes of endovascular thrombectomy versus best medical treatment in children with intracranial arterial occlusion stroke.

Methods: In this prospective registry study, 45 centres in 12 countries across Asia and Australia, Europe, North America, and South America reported functional outcomes for children aged between 28 days and 18 years presenting with arterial ischaemic stroke caused by a large-vessel or medium-vessel occlusion who received either endovascular thrombectomy plus best medical practice or best medical treatment alone.

Polypills in the Management of Cardiovascular Risk-A Perspective.

Cardiovascular disease and cardiovascular risk factors are global healthcare problems, given their high prevalence and the recognized low rates of adequate control despite the abundant body of evidence on different therapeutic options. The World Heart Federation has scrutinized the reasons for poor control of cardiovascular risk factors. Among these reasons, patients' poor adherence to treatment regimens as well as limited rates of evidence-based therapy prescription from healthcare providers play a substantial role in the challenge of cardiovascular risk management.

LLM-AIx: An open source pipeline for Information Extraction from unstructured medical text based on privacy preserving Large Language Models.

In clinical science and practice, text data, such as clinical letters or procedure reports, is stored in an unstructured way. This type of data is not a quantifiable resource for any kind of quantitative investigations and any manual review or structured information retrieval is time-consuming and costly. The capabilities of Large Language Models (LLMs) mark a paradigm shift in natural language processing and offer new possibilities for structured Information Extraction (IE) from medical free text.

Dramatic Impact of Materials Combinations on the Chemical Organization of Core-Shell Nanocrystals: Boosting the Tm Emission above 1600 nm.

This article represents the first foray into investigating the consequences of various material combinations on the short-wave infrared (SWIR, 1000-2000 nm) performance of Tm-based core-shell nanocrystals (NCs) above 1600 nm. In total, six different material combinations involving two different types of SWIR-emitting core NCs (α-NaTmF and LiTmF) combined with three different protecting shell materials (α-NaYF, CaF, and LiYF) have been synthesized. All corresponding homo- and heterostructured NCs have been meticulously characterized by powder X-ray diffraction and electron microscopy techniques.

Development of a Golden Gate Assembly-Based Genetic Toolbox for and Its Application for Engineering Monoterpenoid Biosynthesis.

is a food-grade lactic acid bacterium widely used in the food and beverage industry. Recently, this probiotic organism has been applied as a biofactory for the production of pharmaceutical and food-related compounds, but existing promoters and expression vectors for the genetic engineering of rely on inefficient cloning strategies and are usually not well-characterized. We therefore developed a modular and standardized Golden Gate Assembly-based toolbox for the assembly of shuttle vectors from to .

Integrating digital gait data with metabolomics and clinical data to predict outcomes in Parkinson's disease.

Parkinson's disease (PD) presents diverse symptoms and comorbidities, complicating its diagnosis and management. The primary objective of this cross-sectional, monocentric study was to assess digital gait sensor data's utility for monitoring and diagnosis of motor and gait impairment in PD. As a secondary objective, for the more challenging tasks of detecting comorbidities, non-motor outcomes, and disease progression subgroups, we evaluated for the first time the integration of digital markers with metabolomics and clinical data.

Induction of circulating ABCB1 transcripts under platinum-based chemotherapy indicates poor prognosis and a bone micrometastatic phenotype in ovarian cancer patients.

The drug efflux transporter P-glycoprotein, encoded by the ABCB1 gene, promotes acquired chemoresistance. We explored the presence and clinical relevance of circulating cell-free ABCB1 transcripts (cfABCB1) in ovarian cancer patients (173 longitudinal serum samples from 79 cancer patients) using digital droplet PCR. cfABCB1 were readily detectable at primary diagnosis (median 354 mRNA copies/20 µl serum), paralleled FIGO-stage and predicted surgical outcome (p = 0.

PRAP study-partial versus radical adrenalectomy in hereditary pheochromocytomas.

Objective: Hereditary pheochromocytoma (hPCC) commonly develops bilaterally, causing adrenal insufficiency when standard treatment, radical adrenalectomy (RA), is performed. Partial adrenalectomy (PA) aims to preserve adrenal function, but with higher recurrence rates. This study compares outcomes of PA versus RA in hPCC.

Development and Implementation of a Peer-Teaching Curriculum for Student Assistants.

We have developed a peer-teaching program for student assistants involved in medical education. The offer comprises (1) an inventory of potentially relevant courses offered by other institutions at our university and (2) our own peer-teaching curriculum on pedagogy and teaching methodology. We describe a pilot scheme to implement the curriculum.

Urinary-based detection of MSL, HE4 and CA125 as an additional dimension for predictive and prognostic modelling in ovarian cancer.

Objectives: We have recently described a predictive/prognostic model for ovarian cancer, exploiting commonly available clinico-pathological parameters and the ovarian serum biomarkers mesothelin (MSL), human epididymis protein 4 (HE4) and cancer-antigen 125 (CA125). Considering urine as a prototype non-invasive sample, we investigated whether serum levels of these biomarkers are mirrored in urine and compared their clinical relevance in matched serum urine samples.

Methods: MSL, HE4 and CA125 were quantified in urinary (n=172) and matched serum samples (n=188) from ovarian cancer patients (n=192) using the Lumipulse G chemiluminescent enzyme immunoassay (Fujirebio).