title: [A therapeutic trial of mazindol versus placebo in Duchenne muscular dystrophy. A one-year follow-up study of 14 children].
abstract: A simple-blind therapeutic trial of mazindol (2 mg/d) versus placebo in 14 boys with Duchenne muscular dystrophy, 5 to 13 years old, for 12 months was conducted in order to analyse the efficacy of this drug on the natural history of the disease. Evaluation of muscle strength by manual testing, motor ability by functional testing and timed tests, weight, height, serum CK and pulmonary function were performed every two months. The differences in evolution between groups were significant only for weight and height. However comparison of muscle strength and of vital capacity expressed as percent of predicted at the beginning and at the end of the study suggested a beneficial trend in the mazindol-treated group.
authors.affiliation: Clinique des Maladies du Système nerveux, CHU La Timone, Marseille.
authors.forename: C
authors.initials: C
authors.lastname: Desnuelle
authors.forename: G
authors.initials: G
authors.lastname: Serratrice
authors.forename: V K
authors.initials: VK
authors.lastname: N'guyen
authors.forename: C
authors.initials: C
authors.lastname: Richelme
authors.forename: N
authors.initials: N
authors.lastname: Pinsard
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D002986:Clinical Trials as Topic; D003402:Creatine Kinase / Q000097:blood; D004341:Drug Evaluation; D005500:Follow-Up Studies; D006801:Humans; D007211:Indoles / Q000494:pharmacology*; D008297:Male; D008454:Mazindol / Q000494:pharmacology*; D009129:Muscle Tonus / Q000187:drug effects*; D009136:Muscular Dystrophies / Q000188:drug therapy*; D010919:Placebos; D012129:Respiratory Function Tests
chemical_list: D007211:Indoles; D010919:Placebos; D008454:Mazindol; D003402:Creatine Kinase
vernacular_title: Essai thérapeutique du mazindol contre placebo dans la dystrophie musculaire de Duchenne. Suivi de l'évolution pendant 1 an chez 14 enfants.