title: Corticosteroid therapy for duchenne muscular dystrophy: improvement of psychomotor function.
abstract: BACKGROUND
  Of the numerous clinical trials for Duchenne muscular dystrophy, only the corticosteroid prednisolone has shown potential for temporal improvement in motor ability. In this study, the effects of prednisolone on intellectual ability are examined in 29 cases of Duchenne muscular dystrophy because little information has been reported. And also, motor functions and cardiac functions were evaluated.
  
  
  METHODS
  The treated group was administered prednisolone (0.75 mg/kg) orally on alternate days and the compared with the untreated control group. Gene mutations were investigated. The patients were examined for intelligence quotient adequate for age, brain natriuretic peptide, creatine kinase, and manual muscle testing before treatment and after the period 6 months to 2 years.
  
  
  RESULTS
  Intelligence quotient scores of the treated increased to 6.5 ± 11.9 (mean ± standard deviation) were compared with the controls 2.1 ± 4.9 (P = 0.009). Intelligence quotient scores of the patients with nonsense point mutations improved significantly (21.0 ± 7.9) more than those with deletion or duplication (1.9 ± 9.0; P = 0.015). Motor function, such as time to stand up, of those treated improved significantly and brain natriuretic peptide level was reduced to a normal level after treatment in 15 patients (73%).
  
  
  CONCLUSIONS
  Our results demonstrate the effectiveness of prednisolone in improving intellectual impairment as well as in preserving motor function and brain natriuretic peptide levels. We presume that prednisolone has a read-through effect on the stop codons in the central nervous systems of Duchenne muscular dystrophy because intelligence quotient of point mutation case was improved significantly.
authors.affiliation: Affiliated Field of Genetic Medicine, Division of Biomedical Engineering and Science, Graduate Course of Medicine, Graduate School of Tokyo Women's Medical University, Tokyo, Japan; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan.
authors.forename: Yuko
authors.initials: Y
authors.lastname: Sato
authors.affiliation: Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan.
authors.forename: Akemi
authors.initials: A
authors.lastname: Yamauchi
authors.affiliation: Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan.
authors.forename: Mari
authors.initials: M
authors.lastname: Urano
authors.affiliation: Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan.
authors.forename: Eri
authors.initials: E
authors.lastname: Kondo
authors.affiliation: Affiliated Field of Genetic Medicine, Division of Biomedical Engineering and Science, Graduate Course of Medicine, Graduate School of Tokyo Women's Medical University, Tokyo, Japan; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan. Electronic address: saito@img.twmu.ac.jp.
authors.forename: Kayoko
authors.initials: K
authors.lastname: Saito
mesh_terms: D000305:Adrenal Cortex Hormones / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D003402:Creatine Kinase / Q000097:blood; D005260:Female; D006801:Humans; D007361:Intelligence Tests; D008297:Male; D053580:Muscle Strength / Q000187:drug effects; D020388:Muscular Dystrophy, Duchenne / Q000150:complications* / Q000175:diagnosis / Q000235:genetics; D020097:Natriuretic Peptide, Brain / Q000097:blood; D011239:Prednisolone / Q000031:analogs & derivatives* / Q000627:therapeutic use; D011595:Psychomotor Agitation / Q000188:drug therapy*; D011596:Psychomotor Disorders / Q000188:drug therapy* / Q000209:etiology*; D012189:Retrospective Studies
chemical_list: D000305:Adrenal Cortex Hormones; D020097:Natriuretic Peptide, Brain; D011239:Prednisolone; D003402:Creatine Kinase; C036266:prednylidene
keywords: Duchenne muscular dystrophy; cognition; motor function; prednisolone