Maximal mouth opening in infants and toddlers with spinal muscular atrophy: a prospective controlled study.

Background: Bulbar function is frequently impaired in patients with spinal muscular atrophy (SMA). Although extremely important for the patient's quality of life, it is difficult to address therapeutically. Due to bulbar dysfunction, maximum mouth opening (MMO) is suspected to be reduced in children with SMA.

Identification and characterization of tumor and stromal derived liquid biopsy analytes in pancreatic ductal adenocarcinoma.

Background: The lack of predictive biomarkers contributes notably to the poor outcomes of patients with pancreatic ductal adenocarcinoma (PDAC). Cancer-associated fibroblasts (CAFs) are the key components of the prominent PDAC stroma. Data on clinical relevance of CAFs entering the bloodstream, known as circulating CAFs (cCAFs) are scarce.

Daratumumab plus lenalidomide/dexamethasone in untreated multiple myeloma: analysis of key subgroups of the MAIA study.

In the MAIA study (median follow-up, 56.2 months), daratumumab plus lenalidomide and dexamethasone (D-Rd) significantly improved progression-free survival (PFS) and overall survival versus lenalidomide and dexamethasone (Rd) alone in transplant-ineligible newly diagnosed multiple myeloma (NDMM). In this post hoc analysis of clinically important subgroups in MAIA (median follow-up, 64.

Autologous haematopoietic stem cell transplantation for treatment of multiple sclerosis and neuromyelitis optica spectrum disorder - recommendations from ECTRIMS and the EBMT.

Autologous haematopoietic stem cell transplantation (AHSCT) is a treatment option for relapsing forms of multiple sclerosis (MS) that are refractory to disease-modifying therapy (DMT). AHSCT after failure of high-efficacy DMT in aggressive forms of relapsing-remitting MS is a generally accepted indication, yet the optimal placement of this approach in the treatment sequence is not universally agreed upon. Uncertainties also remain with respect to other indications, such as in rapidly evolving, severe, treatment-naive MS, progressive MS, and neuromyelitis optica spectrum disorder (NMOSD).

Outcomes of mechanical thrombectomy for medium vessel occlusion in acute ischemic stroke patients with ASPECTS 4-5 vs. 6-7: a retrospective, multicenter, and multinational study.

Introduction: Mechanical thrombectomy (MT) efficacy in medium vessel occlusion (MeVO) stroke, particularly in patients with low Alberta Stroke Program Early Computed Tomography Score (ASPECTS), remains less explored.

Methods: This retrospective study analyzed data from 443 AIS patients treated with MT for MeVO and low ASPECTS (4-7) at 37 centers across North America, Asia, and Europe, from September 2017 to July 2021. Patients were categorized into ASPECTS of 4-5 and 6-7.

Relationships of eating behaviors with psychopathology, brain maturation and genetic risk for obesity in an adolescent cohort study.

Unhealthy eating, a risk factor for eating disorders (EDs) and obesity, often coexists with emotional and behavioral problems; however, the underlying neurobiological mechanisms are poorly understood. Analyzing data from the longitudinal IMAGEN adolescent cohort, we investigated associations between eating behaviors, genetic predispositions for high body mass index (BMI) using polygenic scores (PGSs), and trajectories (ages 14-23 years) of ED-related psychopathology and brain maturation. Clustering analyses at age 23 years ( = 996) identified 3 eating groups: restrictive, emotional/uncontrolled and healthy eaters.

Safety and efficacy of the ROCK-2-inhibitor Belumosudil in cGvHD treatment - a retrospective, German-Swiss multicenter real-world data analysis.

Belumosudil is a first in class ROCK2-inhibitor approved by the FDA for the 3rd line treatment of chronic graft-versus-host disease (cGvHD). In this retrospective real-world analysis, we report safety and efficacy data of belumosudil treatment from 5 German/Swiss transplant centers. A total of 33 adult patients (median age 59 years) with moderate (n = 2) or severe (n = 31) cGvHD were treated on individual request due to lack of EMA approval.

Salvage Autologous Transplant in Relapsed Multiple Myeloma: Long-Term Follow-Up of the Phase 3 GMMG ReLApsE Trial.

The multicenter, phase III GMMG ReLApsE trial (EudraCT-No:2009-013856-61) randomized relapsed and/or refractory multiple myeloma (RRMM) patients equally to lenalidomide/dexamethasone (LEN/DEX, 25mg days 1-21/40mg weekly, 4-week cycles) re-induction, salvage high dose chemotherapy (sHDCT, melphalan 200mg/m2), autologous stem cell transplantation (ASCT) and LEN maintenance (10mg/day; transplant arm, n=139) versus continuous LEN/DEX (control arm, n=138). Ninety-four percent of patients had received frontline HDCT/ASCT. We report an updated analysis of survival endpoints with a median follow-up of 99 months.

Minimally invasive versus open surgery for nonfunctioning pancreatic neuroendocrine tumors: a systematic review and meta-analysis.

Introduction: Nonfunctioning pancreatic neuroendocrine tumors (NF-PNETs) have been diagnosed increasingly often but still represent rare pancreatic neoplasms. Surgery is a potentially curative approach for patients with NF-PNETs. In recent years, minimally invasive surgery (MIS) has been applied more frequently for surgical resection of NF-PNETs.

Patient-reported outcome measures for post-COVID-19 condition: a systematic review of instruments and measurement properties.

Objectives: Post-COVID-19 condition (PCC), also referred to as Long COVID, has become an emerging public health issue requiring adequate prevention, treatment and management strategies. Evaluating these strategies from the patients' perspective using patient-reported outcome measures (PROMs) is critical. In this systematic review, we aimed to critically appraise and summarise the quality of existing PROMs for PCC, and to identify PROMs that can be recommended for use in future research.

Hydrolysis of chitin and chitosans by the human chitinolytic enzymes: chitotriosidase, acidic mammalian chitinase, and lysozyme.

Human chitinolytic enzymes trigger growing interest, not only because a wide range of diseases and allergic responses are linked to chitinous components of pathogens, including their interplay with human enzymes, but also due to the increasing use of chitosans in biomedical applications. Here, we present a detailed side-by-side analysis of the only two human chitinases, chitotriosidase and acidic mammalian chitinase, as well as human lysozyme. By analyzing the cleavage of well-characterized chitosan polymers and defined chitin and chitosan oligomers, we report mild processivity and a quantitative subsite preference typical for GH18 chitinases for chitotriosidase and acidic mammalian chitinase.

Nerve transfer of the median flexor pollicis brevis branch to the deep branch of the ulnar nerve for ulnar nerve palsy: a cadaveric feasibility study.

An ulnar nerve (UN) palsy is devastating for hand function, resulting in an intrinsic minus position or claw hand with a loss of pinch grip. Distal nerve transfers facilitate faster reinnervation of hand intrinsic muscles in cases of proximal ulnar nerve lesions. The traditional anterior interosseous nerve (AIN) to UN motor transfer is commonly used, however, this still leads to long reinnervation times for the distal intrinsic muscles, important for the thumb to index pinch grip.

Exploring Tetraselmis chui microbiomes-functional metagenomics for novel catalases and superoxide dismutases.

The focus on microalgae for applications in several fields, e.g. resources for biofuel, the food industry, cosmetics, nutraceuticals, biotechnology, and healthcare, has gained increasing attention over the last decades.

Lutetium-177 PSMA radioligand therapy in taxan-naive first- and second-line metastatic castration resistant prostate cancer after first-line ARPI therapy.

Purpose: Lutetium-177 Prostate-specific membrane antigen (Lu-PSMA) radioligand therapy is EMA-approved for metastatic castration resistant prostate cancer (mCRPC) after androgen receptor pathway inhibition (ARPI) and taxan-based chemotherapy. However, its effect in taxan-naïve patients is under current investigation.

Methods: We relied on the FRAMCAP database to elaborate Lu-PSMA therapy outcomes of progression-free (PFS) and overall (OS) in taxan-naïve mCRPC patients after previous ARPI treatment.

Efficacy and safety of antiviral therapies for the treatment of persistent COVID-19 in immunocompromised patients since the Omicron surge: a systematic review.

Background: Persistent COVID-19 (pCOVID-19) in immunocompromised patients is characterized by unspecific symptoms and pulmonary infiltrates due to ongoing severe acute respiratory syndrome coronavirus-2 (SARS-CoV-2) replication. Treatment options remain unclear, leading to different approaches, including combination therapy and extended durations. The purpose of this study was to assess the efficacy and safety of antiviral therapies for pCOVID-19 in immunocompromised patients since the Omicron surge.

Sacubitril/valsartan reduces proteasome activation and cardiomyocyte area in an experimental mouse model of hypertrophy.

Sacubitril/valsartan (Sac/Val) belongs to the group of angiotensin receptor-neprilysin inhibitors and has been used for the treatment of heart failure (HF) for several years. The mechanisms that mediate the beneficial effects of Sac/Val are not yet fully understood. In this study we investigated whether Sac/Val influences the two proteolytic systems, the ubiquitin-proteasome system (UPS) and the autophagy-lysosomal pathway (ALP), in a mouse model of pressure overload induced by transverse aortic constriction (TAC) and in human induced pluripotent stem cell-derived cardiomyocytes (hiPSC-CMs) treated with endothelin-1 (ET1) serving as a human cellular model of hypertrophy.

Three-year course of clinical high-risk symptoms for psychosis in the community: a latent class analysis.

Aims: Clinical high-risk for psychosis (CHR-P) states exhibit diverse clinical presentations, prompting a shift towards broader outcome assessments beyond psychosis manifestation. To elucidate more uniform clinical profiles and their trajectories, we investigated CHR-P profiles in a community sample.

Methods: Participants ( = 829; baseline age: 16-40 years) comprised individuals from a Swiss community sample who were followed up over roughly 3 years.

Clinical validation of artificial intelligence-based single-subject morphometry without normative reference database.

Background: Single-subject voxel-based morphometry (VBM) is a powerful technique for reader-independent detection of brain atrophy in structural magnetic resonance imaging (MRI) to support the (differential) diagnosis and staging of neurodegenerative diseases in individual patients. However, VBM is sensitive to the MRI scanner platform and details of the acquisition sequence. To mitigate this limitation, we recently proposed and validated a convolutional neural network (CNN)-based VBM which does not rely on a normative reference database.

Acceptance of online therapy for children and adolescents with digital media use disorders: perspectives from child and adolescent psychiatrists and psychotherapists in Germany.

Online therapies have the potential to improve access to psychological services for individuals in need while alleviating the burden on healthcare systems. However, child and adolescent psychiatrists and psychotherapists (CAPPs) rarely integrate these services into their daily practice. This exploratory study investigates CAPPs' acceptance of online therapy, with a focus on treating children and adolescents with digital media use disorders (DMUD).