title: Pharmacological therapies for muscular dystrophies.
abstract: OBJECTIVE
  The study reviews recent advances in pharmacological management of muscular dystrophies. Similarities and differences among the pathophysiology of different forms of muscular dystrophy lead to a broad array of approaches to provide new treatments.
  
  
  RESULTS
  In this review, we include only those muscular dystrophies for which advances have been published in the past year. This represents the 'advancing edge' of a large body of research over more than 20 years. This runs the gamut of new discoveries in symptomatic management to mutation-specific strategies that attempt to correct the root cause of the disorder.
  
  
  CONCLUSIONS
  The field of pharmacological therapies for the muscular dystrophies continues to steadily advance. It is encouraging that research into new therapies is increasingly exploring pharmacological strategies with the potential to ameliorate disease pathology to a clinically significant degree.
authors.affiliation: Division of Neurology, Department of Pediatrics, Children's Hospital of Pittsburgh of UMPC, University of Pittsburgh, Pittsburgh, Pennsylvania 15213, USA.
authors.forename: Hoda
authors.initials: H
authors.lastname: Abdel-Hamid
authors.forename: Paula R
authors.initials: PR
authors.lastname: Clemens
mesh_terms: D000305:Adrenal Cortex Hormones / Q000627:therapeutic use; D047228:Angiotensin II Type 1 Receptor Blockers / Q000627:therapeutic use; D000806:Angiotensin-Converting Enzyme Inhibitors / Q000627:therapeutic use; D000818:Animals; D000889:Anti-Arrhythmia Agents / Q000627:therapeutic use; D000893:Anti-Inflammatory Agents / Q000627:therapeutic use; D001842:Bone and Bones / Q000473:pathology; D018389:Codon, Nonsense / Q000187:drug effects; D006801:Humans; D008801:Mexiletine / Q000627:therapeutic use; D009136:Muscular Dystrophies / Q000188:drug therapy*; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy; D020391:Muscular Dystrophy, Facioscapulohumeral / Q000188:drug therapy; D009223:Myotonic Dystrophy / Q000188:drug therapy; D049411:Utrophin / Q000096:biosynthesis / Q000502:physiology
chemical_list: D000305:Adrenal Cortex Hormones; D047228:Angiotensin II Type 1 Receptor Blockers; D000806:Angiotensin-Converting Enzyme Inhibitors; D000889:Anti-Arrhythmia Agents; D000893:Anti-Inflammatory Agents; D018389:Codon, Nonsense; D049411:Utrophin; D008801:Mexiletine