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Purpose Congenital muscular dystrophy (CMD) comprises a rare group of genetic muscle diseases that present at birth or early during infancy. Two common subtypes of CMD are collagen VI-related muscular dystrophy (COL6-RD) and laminin alpha 2-related dystrophy (LAMA2-RD). Traditional outcome measures in CMD include gross motor and mobility assessments, yet significant motor declines underscore the need for valid upper extremity (UE) motor assessments as a clinical endpoint. This study validated a battery of UE measures in these two CMD subtypes for future clinical trials. Methods For this cross-sectional study, 42 participants were assessed over the same 2–5 day period at the National Institutes of Health Clinical Center (CC). All UE measures were correlated with the Motor Function Measure 32 (MFM32). The battery of UE assessments included the Jebsen Taylor Hand Function Test, Quality of Upper Extremity Skills Test (QUEST), hand held dynamometry, goniometry, and MyoSet Tools. Spearman Rho was used for correlations to the MFM32. Pearson was performed to correlate the Jebsen, QUEST, hand-held dynamometry, goniometry and the MyoSet Tools. Correlations were considered significant at the 0.01 level (2-tailed). Results Significant correlations were found between both the MFM32 and MFM Dimension 3 only (Distal Motor function) and the Jebsen, QUEST, MyoGrip and MyoPinch, elbow flexion/extension ROM and myometry. Additional correlations between the assessments are reported. Conclusions The Jebsen, the Grasp and Dissociated Movements domains of the QUEST, the MyoGrip and the MyoPinch tools, as well as elbow ROM and myometry were determined to be valid and feasible in this population, provided variation in test items, and assessed a range of difficulty in CMD. To move forward, it will be of utmost importance to determine whether these UE measures are reproducible and sensitive to change over time.

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Institutional investment portfolios are currently, and will increasingly be, affected by the risks and opportunities resulting from climate change. This paper contributes new empirical data from 58 in-depth interviews and a global investor survey to explore how climate change is being learnt socially and asocially within the institutional investment industry.This research seeks to identify ways in which the relatively novel concept of 'stranded assets' can be better disseminated to investment professionals. Importantly, both social and asocial learning can affect investment decisions, with some actors usefully providing information via both channels. Better learning, language and leadership within the institutional investment system could facilitate the dissemination of climate and stranded asset discourses among investors, but an imperative to communicate effectively rather than simply communicating more is noted. This paper should interest both investment professionals keen to learn more about the issue and academic researchers seeking to engage investors on these topics.

Institutional investment portfolios are currently, and will increasingly be, affected by the risks and opportunities resulting from climate change. This paper contributes new empirical data from 58 in-depth interviews and a global investor survey to explore how climate change is being learnt socially and asocially within the institutional investment industry. This research seeks to identify ways in which the relatively novel concept of 'stranded assets' can be better disseminated to investment professionals. Importantly, both social and asocial learning can affect investment decisions, with some actors usefully providing information via both channels. Better learning, language and leadership within the institutional investment system could facilitate the dissemination of climate and stranded asset discourses among investors, but an imperative to communicate effectively rather than simply communicating more is noted. This paper should interest both investment professionals keen to learn more about the issue and academic researchers seeking to engage investors on these topics.

BackgroundThere are around 450 rare bone diseases and 10 main types of vasculitis with differing impact on patients and health services. Historically, tests and treatments designed for more common disorders have been applied to both sets of conditions with variable results.Rudy is a recent NIHR funded UK research registry, with strong emphasis on patients' self-reported outcomes, which aims to create a national cohort of patients with rare diseases of the bone, joints and blood vessels. The long term vision is to enable large scale genetics and epidemiology studies and facilitate patient recruitment for clinical trials and biomarker studies.ObjectivesTo analyse the patient self-reported outcome data registered in RUDY up to January 2015.MethodsCollaboration between the National Health Service, Academia, Industry and patients' associations was established. Participants with a clinical diagnosis of a rare disorder of the bones, joints or blood vessels with a prevalence of less than 1:5000 were recruited using a web based platform. The diagnosis and demographics were registered and one set of questionnaires was offered for completion to all patients.ResultsThere were 133 patients (71% females) registered; mean age 44.6±18.1 years [3-81] with 10 patients <18 years. Rare bone or joint disorders were more frequent (65%), particularly fibrous dysplasia. The most common diagnosis of vasculitis was granulomatosis with polyangiitis (GPA) (49%) [Table].On line assessments of general health were performed: EQ5D-5L was completed by 56% of patients with a mean score of 2.2±1.2 in mobility, 1.6±0.9 in self-care, 2.1±1.1 in usual activities, 2.6±0.9 in pain/discomfort and 1.8±0.8 in anxiety/depression (range 0-4, best to worst) and 68.4±19.9 general health (range 0-100, worst to best); SF-36 by 53% of patients with a mean score of 47.1±40.3 in physical functioning, 29.3±45.5 in limitations due to physical health, 58.1±49.3 in limitations due to emotional problems, 40.6±27.3 in energy/ fatigue, 69.2±26.1 in emotional wellbeing, 61.3±29.5 in social functioning, 53.9±28.1 in pain and 40.2±30.0 in general health (range 0-100, worst to best); Nottingham ADL score by 59% of patients with a mean score of 19.1±3.9 (range 0-22, worst to best); and Pittsburgh Sleep Quality Index by 56% of patients with a mean score of 9.8±4.0 (range 0-21, best to worst). Pain was further assessed through the Pain detect and McGill pain questionnaires in 53% of patients. Paediatric participants have recently been asked to fill the Childhood Health Assessment, Paediatric Quality of Life and Wong-Baker faces' questionnaires. Other disease specific questionnaires, prospective evaluation of the outcome measures assessed, and the construction of a bone fracture map and a family tree for relatives to participate in are being developed.ConclusionsThe RUDY project provides an easy way to collect valuable data and facilitate research on patients with rare diseases of bone and in the vasculitides. A significant proportion of patients reported impairment in several do...

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