Monitoring daily physical activity of upper extremity in young and adolescent boys with Duchenne muscular dystrophy:A pilot study

Introduction Accelerometry of the upper extremity (UE) potentially provides information on the extent of activities in daily life in patients with Duchenne muscular dystrophy (DMD). The objective of this study is to evaluate the validity of home measurements of UE accelerometry. Methods This was a cross-sectional study in 16 patients with DMD (aged 7-17 years). Patients were monitored for 1 to 3 days with two accelerometers on the UE and one accelerometer on the wheelchair. Results The mean intensity of activity and the mean frequency of transfers of arm elevation from low to middle were approximately twofold higher in patients with a Brooke scale score of 1 or 2 than in patients with a Brooke scale score of 3 or 4. Correlations with the Performance of Upper Limb scale score were high for intensity and for the total frequency of arm elevations per hour. Discussion Intensity, percentage of time in middle orientation, and frequency of transfers of the upper arm correlated well with functional measurements

Regulation of Drosophila Brain Wiring by Neuropil Interactions via a Slit-Robo-RPTP Signaling Complex

The axonal wiring molecule Slit and its Round-About (Robo) receptors are conserved regulators of nerve cord patterning. Robo receptors also contribute to wiring brain circuits. Whether molecular mechanisms regulating these signals are modified to fit more complex brain wiring processes is unclear. We investigated the role of Slit and Robo receptors in wiring Drosophila higher-order brain circuits and identified differences in the cellular and molecular mechanisms of Robo/Slit function. First, we find that signaling by Robo receptors in the brain is regulated by the Receptor Protein Tyrosine Phosphatase RPTP69d. RPTP69d increases membrane availability of Robo3 without affecting its phosphorylation state. Second, we detect no midline localization of Slit during brain development. Instead, Slit is enriched in the mushroom body, a neuronal structure covering large areas of the brain. Thus, a divergent molecular mechanism regulates neuronal circuit wiring in the Drosophila brain, partly in response to signals from the mushroom body

Eye movements in patients with Whiplash Associated Disorders: a systematic review

textabstractBackground: Many people with Whiplash Associated Disorders (WAD) report problems with vision, some of which may be due to impaired eye movements. Better understanding of such impaired eye movements could improve diagnostics and treatment strategies. This systematic review surveys the current evidence on changes in eye movements of patients with WAD and explains how the oculomotor system is tested. Methods: Nine electronic data bases were searched for relevant articles from inception until September 2015. All studies which investigated eye movements in patients with WAD and included a healthy control group were screened for inclusion. Qualifying studies were retrieved and independently assessed for methodological quality using the Methodology Checklists provided by the Scottish Intercollegiate Guidelines Network. Results: Fourteen studies out of 833 unique hits were included. Ten studies reported impaired eye movements in patients with WAD and in four studies no differences compared to healthy controls were found. Different methods of eye movement examination were used in the ten studies: in five studies, the smooth pursuit neck torsion test was positive, in two more the velocity and stability of head movements during eye-coordination tasks were decreased, and in another three studies the cervico-ocular reflex was elevated. Conclusions: Overall the reviewed studies show deficits in eye movement in patients with WAD, but studies and results are varied. When comparing the results of the 14 relevant publications, one should realise that there are significant differences in test set-up and patient population. In the majority of studies patients show altered compensatory eye movements and smooth pursuit movements which may impair the coordination of head and eyes

Motor Learning in Children with Neurofibromatosis Type I

The aim of this study was to quantify the frequently observed problems in motor control in Neurofibromatosis type 1 (NF1) using three tasks on motor performance and motor learning. A group of 70 children with NF1 was compared to age-matched controls. As expected, NF1 children showed substantial problems in visuo-motor integration (Beery VMI). Prism-induced hand movement adaptation seemed to be mildly affected. However, no significant impairments in the accuracy of simple eye or hand movements were observed. Also, saccadic eye movement adaptation, a cerebellum dependent task, appeared normal. These results suggest that the motor problems of children with NF1 in daily life are unlikely to originate solely from impairments in motor learning. Our findings, therefore, do not support a general dysfunction of the cerebellum in children with NF1

Clinical, methodology, and patient/carer expert advice in pediatric drug development by conect4children.

Many medicines are used "off-label" in children outside the terms of the license. Feasible pediatric clinical trials are a challenge to design. Conect4children (c4c) is an Innovative Medicines Initiative project to set up a pan-European pediatric clinical trial network aiming to facilitate the development of new medicines for children. To optimize pediatric trial development by promoting innovative trial design, c4c set up a European multidisciplinary advice service, including the voice of young patients and families, tailored to industry and academia. A network of experts was established to provide multidisciplinary advice to trial sponsors. Experts were selected to join clinical and innovative methodology expert groups. A patient and public involvement (PPI) database, to include the expert opinion of patients and parents/carers was formed. A stepwise process was developed: (1) sponsors contact c4c, (2) scoping interview takes place, (3) ad hoc advice group formed, (5) advice meeting held, and (6) advice report provided. Feedback on the process was collected. Twenty-four clinical and innovative methodology expert groups (>400 experts) and a PPI database of 135 registrants were established. As of September 30, 2022, 36 advice requests were received, with 25 requests completed. Clinical and methodology experts and PPI representatives participated in several advice requests. Sponsors appreciated the advice quality and the multidisciplinary experts from different countries, including experts not known before. Experts and PPI participants were generally satisfied with the process. The c4c project has shown successful proof of concept for a service that presents a new framework to plan innovative and feasible pediatric trials

Development and evaluation of a patient centered cardiovascular health education program for insured patients in rural Nigeria (QUICK - II)

<p>Abstract</p> <p>Background</p> <p>In Sub Saharan Africa, the incidence of hypertension and other modifiable cardiovascular risk factors is growing rapidly. Poor adherence to prescribed prevention and treatment regimens by patients can compromise treatment outcomes. Patient-centered cardiovascular health education is likely to improve shortcomings in adherence. This paper describes a study that aims to develop a cardiovascular health education program for patients participating in a subsidized insurance plan in Nigeria and to evaluate the applicability and effectiveness in patients at increased risk for cardiovascular disease.</p> <p>Methods/Design</p> <p><it>Design: </it>The study has two parts. Part <it>1 </it>will develop a cardiovascular health education program, using qualitative interviews with stakeholders. Part <it>2 </it>will evaluate the effectiveness of the program in patients, using a prospective (pre-post) observational design.</p> <p><it>Setting: </it>A rural primary health center in Kwara State, Nigeria.</p> <p><it>Population: </it>For part 1: 40 patients, 10 healthcare professionals, and 5 insurance managers. For part 2: 150 patients with uncontrolled hypertension or other cardiovascular risk factors after one year of treatment.</p> <p><it>Intervention: </it>Part <it>2</it>: patient-centered cardiovascular health education program.</p> <p><it>Measurements: </it>Part 1: Semi-structured interviews to identify stakeholder perspectives. Part 2: Pre- and post-intervention assessments including patients' demographic and socioeconomic data, blood pressure, body mass index and self-reporting measures on medication adherence and perception of care. Feasibility of the intervention will be measured using process data.</p> <p><it>Outcomes: </it>For program development (part 1): overview of healthcare professionals' perceptions on barriers and facilitators to care, protocol for patient education, and protocol implementation plan.</p> <p>For program evaluation (part 2): changes in patients' scores on adherence to medication and life style changes, blood pressure, and other physiological and self-reporting measures at six months past baseline.</p> <p><it>Analysis: </it>Part 1: content analytic technique utilizing MAXQDA software. Part 2: univariate and multilevel analysis to assess outcomes of intervention.</p> <p>Discussion</p> <p>Diligent implementation of patient-centered education should enhance adherence to cardiovascular disease prevention and management programs in low income countries.</p> <p>Trial Registration</p> <p><a href="http://www.controlled-trials.com/ISRCTN47894401">ISRCTN47894401</a></p