Adult patients with DMD develop progressive impairment, due to respiratory, orthopaedic and general medical factors. However, the particular areas of difficulty in this study often reflected inadequate and poorly directed social and medical support, illustrating the need for improvements in the structure, co-ordination and breadth of rehabilitation services for adult patients with DMD.

Early clinical trials of therapies to treat Duchenne muscular dystrophy (DMD), a fatal genetic X-linked pediatric disease, have been designed based on the limited understanding of natural disease progression and variability in clinical measures over different stages of the continuum of the disease. The objective was to inform the design of DMD clinical trials by developing a disease progression modelbased clinical trial simulation (CTS) platform based on measures commonly used in DMD trials. Data were integrated from past studies through the Duchenne Regulatory Science Consortium founded by the Critical Path Institute (15 clinical trials and studies, 1505 subjects, 27,252 observations). Using a nonlinear mixedeffects modeling approach, longitudinal dynamics of five measures were modeled (NorthStar Ambulatory Assessment, forced vital capacity, and the velocities of the following three timed functional tests: time to stand from supine, time to climb 4 stairs, and 10 meter walk-run time). The models were validated on external data sets and captured longitudinal changes in the five measures well, including both early disease when function improves as a result of growth and development and the decline in function in later stages. The models can be used in the CTS platform to perform trial simulations to optimize the selection of inclusion/ exclusion criteria, selection of measures, and other trial parameters. The data sets and models have been reviewed by the US Food and Drug Administration and the European Medicines Agency; have been accepted into the Fit-for-Purpose and Qualification for Novel Methodologies pathways, respectively; and will be submitted for potential endorsement by both agencies.

BackgroundFalls are a leading cause of injury-related hospitalizations among adults aged 65 years and older and may result in social isolation.ObjectiveTo summarise evidence on falls and subsequent social isolation and/or loneliness in older adults through a scoping review.Eligibility criteriaStudies were eligible for inclusion if the population had a mean age of 60 years or older, they examined falls and subsequent social isolation, loneliness, fear of falling or risk factors and were primary studies (eg, experimental, quasi-experimental, observational and qualitative).Sources of evidenceMEDLINE, CINAHL, Embase, Ageline and grey literature from inception until 11 January 2021.Charting methodsA screening and charting form was developed and pilot-tested. Subsequently, two reviewers screened citations and full-text articles, and charted the evidence.ResultsAfter screening 4993 citations and 304 full-text articles, 39 studies were included in this review. Participants had a history of falling (range: 11% to 100%). Most studies were conducted in Europe (44%) and North America (33%) and were of the cross-sectional study design (66.7%), in the community (79%). Studies utilised 15 different scales. Six studies examined risk factors for social isolation and activity restriction associated with fear of falling. Six studies reported mental health outcomes related to falls and subsequent social isolation.ConclusionsConsistency in outcome measurement is recommended, as multiple outcomes were used across the included studies. Further research is warranted in this area, given the ageing population and the importance of falls and social isolation to the health of older adults.Scoping review registration number10.17605/OSF.IO/2R8HM.

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Introduction In the past decade, investigations of the relationship between sleep duration and eating behaviours have been emerging; however, a formal synthesis of the literature focussed on adolescent populations has not yet been conducted. We conducted a scoping review of the literature examining the relationship between sleep duration and eating behaviours in adolescents. Gaps in the research and directions for future research were identified based on the findings. Methods A systematic search was employed on four research databases: PubMed, PsycInfo, CINAHL and Scopus; relevant grey literature was also reviewed. Studies that reported on the relationship between sleep duration and eating behaviours among high school–aged adolescents were included in the review. Data were extracted, charted and synthesized into a narrative. Consistent with the purpose of a scoping review, the methodological quality of the studies was not appraised. Stakeholders were consulted to validate the findings and provide insight into the interpretation and identification of pressing gaps in the research that remain to be addressed. Results In total, 61 studies published between 2006 and 2021 met the criteria for review. Existing research focussed heavily on examining sleep duration in relation to intake of food from certain food groups, beverages and processed foods, and relied on a population study design, cross-sectional analyses and self-report measures. Conclusion Future research is needed to understand the link between sleep duration and eating-related cognition, eating contexts and disordered eating behaviours in order to better understand how ensuring sufficient sleep among adolescents can be leveraged to support healthier eating practices and reduce diet-related risks.