Outcome Measures for Pediatric Spinal Muscular Atrophy
نویسندگان
چکیده
منابع مشابه
Outcome measures for pediatric spinal muscular atrophy.
BACKGROUND Spinal muscular atrophy (SMA) is a genetic disease of the anterior horn cell with a frequency of 8 per 100 000 live births and a high rate of mortality during infancy. The American Spinal Muscular Atrophy Randomized Trials (AmSMART) Group is an organization of 5 centers formed to perform clinical trials in children with SMA. OBJECTIVE To devise reliable methods to measure strength,...
متن کاملClinical outcome measures in spinal muscular atrophy.
Spinal muscular atrophy is one of the most devastating neurological diseases of childhood. Affected infants and children suffer from often severe muscle weakness caused by degeneration of lower motor neurons in the spinal cord and brainstem. Identification of the causative genetic mutation in most cases has resulted in development of potential treatment strategies. To test these new drugs, clin...
متن کاملRasch analysis of clinical outcome measures in spinal muscular atrophy.
INTRODUCTION Trial design for SMA depends on meaningful rating scales to assess outcomes. In this study Rasch methodology was applied to 9 motor scales in spinal muscular atrophy (SMA). METHODS Data from all 3 SMA types were provided by research groups for 9 commonly used scales. Rasch methodology assessed the ordering of response option thresholds, tests of fit, spread of item locations, res...
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Spinal muscular atrophy (SMA) is a genetic disorder which affect nervous system and is characterized with progressive distal motor neuron weakness. The survival motor neuron (SMN) protein level reduces in patients with SMA. Two different genes code survival motor neuron protein in human genome. Skeletal and intercostal muscles denervation lead to weakness, hypotony, hyporeflexia, respiratory fa...
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ژورنال
عنوان ژورنال: Archives of Neurology
سال: 2002
ISSN: 0003-9942
DOI: 10.1001/archneur.59.9.1445