Evaluation of the muscle strength and motor ability in children with spinal muscle atrophy type II and III treated with valproic acid / Avaliação da força muscular e da habilidade motora das crianças com amiotrofia espinhal progressiva do tipo II e III medicadas com ácido valpróico

AUTOR(ES)
DATA DE PUBLICAÇÃO

2009

RESUMO

Spinal Muscular Atrophy (SMA) is an autosomal recessive disorder that affects the spinal motoneurons, resulting in hypotonia and muscle weakness. The knowledge of the molecular mechanism of SMA has originated new researches including clinical trials with pharmacological agents that increase SMN2 protein level. Many drugs with this action are being tested with the aim of finding a possible treatment for this severe disease. The valproic acid (VA), a well-known drug used to treat epilepsy has the property of activating the SMN2 gene promoter and then to increase SMN2 protein level. Since there isnt an uniform system for the clinical evaluation of the treatment results, we selected a set of easy, fast and already validated methods to evaluate if the VA is effective to stabilize or improve the motor function in patients with SMA. The selected methods were: Medical Research Council scale (MRC) to muscular strength; Hammersmith motor ability score to motor ability; goniometry of the main joints; time to walk when possible; Barthel índex for daily activities, and a questionnaire to verify the types of physiotherapy and hydrotherapy in use. Twenty two patients with SMA II and III, aged between 2 and 19 years, and treated with VA were evaluated every three months during the period of one year; the first evaluation occurred immediately before the onset of the treatment. The results of the tests demonstrated that along the period of 12 months the patients didnt gain muscle strength but improved their motor ability, that can be considered a positive result. Children aged six years or younger had a higher gain in motor ability along the period of the study.

ASSUNTO(S)

methods of evaluation amiotrofia espinhal spinal muscular atrophy medical research council (mrc) métodos de avaliação hammersmith motor ability score (hmas) hammersmith motor ability score (hmas) medical research council (mrc) Ácido valpróico valproic acid

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