The North Star ambulatory assessment (NSAA) is a functional motor outcome measure in Duchenne muscular dystrophy (DMD), widely used in clinical trials and natural history studies, as well as in... Show moreThe North Star ambulatory assessment (NSAA) is a functional motor outcome measure in Duchenne muscular dystrophy (DMD), widely used in clinical trials and natural history studies, as well as in clinical practice. However, little has been reported on the minimal clinically important difference (MCID) of the NSAA. The lack of established MCID estimates for NSAA presents challenges in interpreting the significance of the results of this outcome measure in clinical trials, natural history studies and clinical practice. Combining statistical approaches and patient perspectives, this study estimated MCID for NSAA using distribution-based estimates of 1/3 standard deviation (SD) and standard error of measurement (SEM), an anchor-based approach, with six-minute walk distance (6MWD) as the anchor, and evaluation of patient and parent perception using participant-tailored questionnaires. The MCID for NSAA in boys with DMD aged 7 to 10 years based on 1/3 SD ranged from 2.3–2.9 points, and that on SEM ranged from 2.9–3.5 points. Anchored on the 6MWD, the MCID for NSAA was estimated as 3.5 points. When the impact on functional abilities was considered using participant response questionnaires, patients and parent perceived a complete loss of function in a single item or deterioration of function in one to two items of the assessment as an important change. Our study examines MCID estimates for total NSAA scores using multiple approaches, including the impact of patient and parent perspective on within scale changes in items based on complete loss of function and deterioration of function, and provides new insight on evaluation of differences in these widely used outcome measure in DMD. Show less
The Caucus for a New Political Science (CNPS), emerged from a meeting of graduate students at the 1967 convention of the American Political Science Association. It is based on a demand for a new... Show moreThe Caucus for a New Political Science (CNPS), emerged from a meeting of graduate students at the 1967 convention of the American Political Science Association. It is based on a demand for a new relevance in political science. Political science is considered irrelevant if it is uncritical of society and assumes the values and social priorities of corrupt bureaucracies, powerful elites or unjust social practices. The ’established’ political science profession is accused of justifying everything in the American political system as unique and workable and of condeming attempts to change it. Stability, rather than change, is the highest value, and conflict may be resolved peacefully and fairly within the existing system. The new conservatives desire to avoid major issues in favor of trivialities is mentioned. Behavioralism, emphasizing the fact/value dichotomy and preoccupied with questions of method and model-building, supports these conservative tendencies. Political scientists have become ideologues of the government. Professionalism and its rewards are determined less by the values and ethics of the discipline than by the values and ethics of the social and political status quo. A radical political science is needed to give more relevance to the political science profession. A radical political science can be defined as an attempt to use the tools which the discipline has created in order to solve the problems which society has created. There are three prospective lines of development for a radical political science: (i) a primarily theoretical one — the development of new paradigms for research and political analysis or new modes of inquiry; (2) the politization and democratization of the APSA and the reform of the discipline, teaching, curricula — with the CNPS as the pinwheel for reform; (3) the creation of a research-action political science focusing on criticism of American institutions and analyses of alternative social priorities. Show less
