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In addition, we could show coherent multisite results for a sophisticated CEST method, highlighting the benefits regarding protocol sharing and reproducibility.

With Pulseq-CEST, we provide a straightforward approach to standardize, share, simulate, and measure different CEST preparation schemes, which are inherently completely defined.

With Pulseq-CEST, we provide a straightforward approach to standardize, share, simulate, and measure different CEST preparation schemes, which are inherently completely defined.

In this study we report the results of a phase Ib/IIa, open-label, multiple ascending-dose trial of domagrozumab, a myostatin inhibitor, in patients with fukutin-related protein (FKRP)-associated limb-girdle muscular dystrophy.

Nineteen patients were enrolled and assigned to one of three dosing arms (5, 20, or 40 mg/kg every 4 weeks). After 32 weeks of treatment, participants receiving the lowest dose were switched to the highest dose (40 mg/kg) for an additional 32 weeks. An extension study was also conducted. The primary endpoints were safety and tolerability. Secondary endpoints included muscle strength, timed function testing, pulmonary function, lean body mass, pharmacokinetics, and pharmacodynamics. see more As an exploratory outcome, muscle fat fractions were derived from whole-body magnetic resonance images.

Serum concentrations of domagrozumab increased in a dose-dependent manner and modest levels of myostatin inhibition were observed in both serum and muscle tissue. The most frequently occurring adverse events were injuries secondary to falls. There were no significant between-group differences in the strength, functional, or imaging outcomes studied.

We conclude that, although domagrozumab was safe in patients in limb-girdle muscular dystrophy type 2I/R9, there was no clear evidence supporting its efficacy in improving muscle strength or function.

We conclude that, although domagrozumab was safe in patients in limb-girdle muscular dystrophy type 2I/R9, there was no clear evidence supporting its efficacy in improving muscle strength or function.Myofibroblasts, renowned for their contractility and extracellular matrix production, are widely considered the key effector cells for nearly all scars resulting from tissue repair processes, ranging from normal scars to extreme fibrosis. For example, it is often assumed that myofibroblasts underpin the characteristics of keloid scars, which are debilitating pathological skin scars lacking effective treatments because of a poor understanding of the disease mechanisms. Here, we present primary and published transcriptional and histological evidence that myofibroblasts are not consistently present in primary keloid lesions, and when alpha-smooth muscle actin (αSMA)-positive cells are detected, they are not greater in number or expressing more αSMA than in normal or hypertrophic scars. In conclusion, keloid scars do not appear to require αSMA-positive myofibroblasts; continuing to consider keloids on a quantitative spectrum with normal or hypertrophic scars, with αSMA serving as a biomarker of disease severity, is hindering advancement of understanding and therapy development.

To develop an evidence-based, culturally tailored, diabetes self-management education and support programme for Black-British adults, called Healthy Eating and Active Lifestyles for Diabetes (HEAL-D), using participatory methods to engage key stakeholders in the intervention design process.

Black-British adults living with type 2 diabetes, healthcare professionals and community leaders were engaged in an intervention development study. The intervention structure, format, content and delivery were developed through three phases of participatory research Phase 1, formative research, involved focus groups and interviews; interactive co-development workshops were conducted in Phase 2; and Phase 3 focused on materials development.

In Phase 1, focus groups and interviews identified the importance of nurturing collectivism, a reliance on informal sources of information/advice, barriers to attending appointments associated with competing priorities of work, travel and carer commitments, and a preference for dirment of healthcare interventions that are sensitive to the needs of service users and providers.Metabolic syndrome (MetS) is characterized by insulin resistance, high blood pressure/sugar, dyslipidemia, and obesity. Whether MetS and its components affect the development of Behçet's disease (BD) remains unclear. This study was performed to investigate the associations between metabolic syndrome and risk of BD using nationwide population data. We conducted a retrospective cohort study of 10 505 818 Korean subjects who received health checkups in 2009-2012. Patients were classified into a MetS and its components group and were followed-up until 2016 for new-onset BD. A Cox proportional hazards model was used to assess the independent or synergistic effects of MetS and its components on the risk of incident BD. Compared to subjects without MetS components, the hazard ratio (HR) for development of BD in patients with MetS was 0.874 (95% confidence interval [CI] 0.819-0.933) and this association was more prominent when all components of MetS were present (HR = 0.675, 95% CI = 0.571-0.798). Subjects with low high density lipoprotein (HDL) has a significantly increased risk of the development of BD (HR = 1.51, 95% CI = 1.4-1.594) compared to controls. This study showed that the incidence of Behçet's disease was reduced in subjects with MetS. Moreover, the presence of MetS components, with the exception of HDL, was negatively related to the development of BD.

Pre-emptive kidney transplantation (PKT) is generally considered the optimal treatment for kidney failure as it minimises dialysis-associated morbidity and mortality and is associated with improved allograft survival. This study aimed to determine rates of paediatric PKT in New Zealand, identify barriers to PKT and consider potential interventions to influence future rates of pre-emptive transplantation.

Children commencing kidney replacement therapy between 2005 and 2017 in New Zealand were included. Descriptive analysis considered those referred late (referral <3months prior to kidney replacement therapy initiation) or early based on referral timing to paediatric nephrology. Additional analysis compared characteristics of children receiving dialysis versus pre-emptive transplant as their first mode of kidney replacement therapy.

PKT occurred in 15 of 90 children (17%). One-third of all patients were referred late. No late referrals received a pre-emptive transplant. Pre-emptively transplanted children were referred younger (median age 0.

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