title: Clinical aspects of patients with sarcoglycanopathies under steroids therapy.
abstract: Patients with sarcoglycanopathies, which comprise four subtypes of autosomal recessive limb-girdle muscular dystrophies, usually present with progressive weakness leading to early loss of ambulation and premature death, and no effective treatment is currently available.
  
  
  OBJECTIVE
  To present clinical aspects and outcomes of six children with sarcoglycanopathies treated with steroids for at least one year.
  
  
  METHODS
  Patient files were retrospectively analyzed for steroid use.
  
  
  RESULTS
  Stabilization of muscle strength was noted in one patient, a slight improvement in two, and a slight worsening in three. In addition, variable responses of forced vital capacity and cardiac function were observed.
  
  
  CONCLUSIONS
  No overt clinical improvement was observed in patients with sarcoglycanopathies under steroid therapy. Prospective controlled studies including a larger number of patients are necessary to determine the effects of steroids for sarcoglycanopathies.
authors.affiliation: Departamento de Neurologia, Faculdade de Medicina, Universidade de São Paulo, São Paulo, SP, Brazil.
authors.forename: Marco A V
authors.initials: MA
authors.lastname: Albuquerque
authors.affiliation: Departamento de Neurologia, Faculdade de Medicina, Universidade de São Paulo, São Paulo, SP, Brazil.
authors.forename: Osório
authors.initials: O
authors.lastname: Abath-Neto
authors.affiliation: Departamento de Neurologia, Faculdade de Medicina, Universidade de São Paulo, São Paulo, SP, Brazil.
authors.forename: Jéssica R
authors.initials: JR
authors.lastname: Maximino
authors.affiliation: Departamento de Neurologia, Faculdade de Medicina, Universidade de São Paulo, São Paulo, SP, Brazil.
authors.forename: Gerson
authors.initials: G
authors.lastname: Chadi
authors.affiliation: Departamento de Neurologia, Faculdade de Medicina, Universidade de São Paulo, São Paulo, SP, Brazil.
authors.forename: Edmar
authors.initials: E
authors.lastname: Zanoteli
authors.affiliation: Departamento de Neurologia, Faculdade de Medicina, Universidade de São Paulo, São Paulo, SP, Brazil.
authors.forename: Umbertina C
authors.initials: UC
authors.lastname: Reed
mesh_terms: D002648:Child; D005260:Female; D005938:Glucocorticoids / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D011239:Prednisolone / Q000627:therapeutic use*; D011282:Pregnenediones / Q000627:therapeutic use*; D012189:Retrospective Studies; D058088:Sarcoglycanopathies / Q000188:drug therapy*; D016896:Treatment Outcome
chemical_list: D005938:Glucocorticoids; D011282:Pregnenediones; D011239:Prednisolone; C021988:deflazacort