28 research outputs found

    The second physical therapy summit on global health: developing an action plan to promote health in daily practice and reduce the burden of non-communicable diseases

    Get PDF
    Based on indicators that emerged from The First Physical Therapy Summit on Global Health (2007), the Second Summit (2011) identified themes to inform a global physical therapy action plan to integrate health promotion into practice across the World Confederation for Physical Therapy (WCPT) regions. Working questions were: (1) how well is health promotion implemented within physical therapy practice; and (2) how might this be improved across five target audiences (i.e. physical therapist practitioners, educators, researchers, professional body representatives, and government liaisons/consultants). In structured facilitated sessions, Summit representatives (n=32) discussed: (1) within WCPT regions, what is working and the challenges; and (2) across WCPT regions, what are potential directions using World CaféTM methodology. Commonalities outweighed differences with respect to strategies to advance health-focused physical therapy as a clinical competency across regions and within target audiences. Participants agreed that health-focused practice is a professional priority, and a strategic action plan was needed to develop it as a clinical competency. The action plan and recommendations largely paralleled the principles and objectives of the World Health Organization's non-communicable diseases action plan. A third Summit planned for 2015 will provide a mechanism for follow-up to evaluate progress in integrating health-focused physical therapy within the profession.info:eu-repo/semantics/acceptedVersio

    Ataluren treatment of patients with nonsense mutation dystrophinopathy

    Get PDF
    Introduction: Dystrophinopathy is a rare, severe muscle disorder, and nonsense mutations are found in 13% of cases. Ataluren was developed to enable ribosomal readthrough of premature stop codons in nonsense mutation (nm) genetic disorders. Methods: Randomized, double‐blind, placebo‐controlled study; males ≄5 years with nm‐dystrophinopathy received study drug orally 3 times daily, ataluren 10, 10, 20 mg/kg (N = 57); ataluren 20, 20, 40 mg/kg (N = 60); or placebo (N = 57) for 48 weeks. The primary endpoint was change in 6‐Minute Walk Distance (6MWD) at Week 48. Results: Ataluren was generally well tolerated. The primary endpoint favored ataluren 10, 10, 20 mg/kg versus placebo; the week 48 6MWD Δ = 31.3 meters, post hoc P = 0.056. Secondary endpoints (timed function tests) showed meaningful differences between ataluren 10, 10, 20 mg/kg, and placebo. Conclusions: As the first investigational new drug targeting the underlying cause of nm‐dystrophinopathy, ataluren offers promise as a treatment for this orphan genetic disorder with high unmet medical need

    Top 10 Research Questions Related to Physical Activity and Multiple Sclerosis

    No full text
    An estimated 2.5 million people worldwide are living with multiple sclerosis (MS), and this disease may be increasing in prevalence. MS is a disease of the central nervous system that is associated with heterogeneous symptoms and functional consequences, and the current firstline disease-modifying therapies often become ineffective later in the disease. There is increasing evidence for the benefits of physical activity (PA) in people with MS, but this population is generally physically inactive and sedentary. We proposed 10 research questions to guide future research on PA and MS: (1) Is PA an MS disease-modifying behavior? (2) What are the benefits of PA among people with MS? (3) What is the optimal PA prescription for people with MS? (4) What are the safety issues with PA in people with MS? (5) What characteristics of people with MS modify the benefits of PA? (6) What variables explain participation in PA among people with MS? (7) What are effective behavioral interventions for PA change in people with MS? (8) How do we translate PA research into clinical MS practice? (9) What is the role of sedentary behavior in people with MS? And (10) what is the optimal measurement of PA in people with MS? These questions are critical for informing our understanding of the short- and long-term consequences of PA in MS as well as for identifying approaches for promoting and sustaining PA in MS. Addressing these questions may greatly improve the lives of people with this chronic diseas

    THE 6-MINUTE WALK TEST AND OTHER ENDPOINTS IN DUCHENNE MUSCULAR DYSTROPHY: LONGITUDINAL NATURAL HISTORY OBSERVATIONS OVER 48 WEEKS FROM A MULTICENTER STUDY

    Get PDF
    INTRODUCTION: Duchenne muscular dystrophy (DMD) subjects ≄5 years with nonsense mutations were followed for 48 weeks in a multicenter, randomized, double-blind, placebo-controlled trial of ataluren. Placebo arm data (N = 57) provided insight into the natural history of the 6-minute walk test (6MWT) and other endpoints. METHODS: Evaluations performed every 6 weeks included the 6-minute walk distance (6MWD), timed function tests (TFTs), and quantitative strength using hand-held myometry. RESULTS: Baseline age (≄7 years), 6MWD, and selected TFT performance are strong predictors of decline in ambulation (Δ6MWD) and time to 10% worsening in 6MWD. A baseline 6MWD of <350 meters was associated with greater functional decline, and loss of ambulation was only seen in those with baseline 6MWD <325 meters. Only 1 of 42 (2.3%) subjects able to stand from supine lost ambulation. CONCLUSION: Findings confirm the clinical meaningfulness of the 6MWD as the most accepted primary clinical endpoint in ambulatory DMD trials
    corecore