title: Muscle-Derived Proteins as Serum Biomarkers for Monitoring Disease Progression in Three Forms of Muscular Dystrophy.
abstract: BACKGROUND
  Identifying translatable, non-invasive biomarkers of muscular dystrophy that better reflect the disease pathology than those currently available would aid the development of new therapies, the monitoring of disease progression and the response to therapy.
  
  
  OBJECTIVE
  The goal of this study was to evaluate a panel of serum protein biomarkers with the potential to specifically detect skeletal muscle injury.
  
  
  METHODS
  Serum concentrations of skeletal troponin I (sTnI), myosin light chain 3 (Myl3), fatty acid binding protein 3 (FABP3) and muscle-type creatine kinase (CKM) proteins were measured in 74 Duchenne muscular dystrophy (DMD), 38 Becker muscular dystrophy (BMD) and 49 Limb-girdle muscular dystrophy type 2B (LGMD2B) patients and 32 healthy controls.
  
  
  RESULTS
  All four proteins were significantly elevated in the serum of these three muscular dystrophy patient populations when compared to healthy controls, but, interestingly, displayed different profiles depending on the type of muscular dystrophy. Additionally, the effects of patient age, ambulatory status, cardiac function and treatment status on the serum concentrations of the proteins were investigated. Statistical analysis revealed correlations between the serum concentrations and certain clinical endpoints including forced vital capacity in DMD patients and the time to walk ten meters in LGMD2B patients. Serum concentrations of these proteins were also elevated in two preclinical models of muscular dystrophy, themdxmouse and the golden-retriever muscular dystrophy dog.
  
  
  CONCLUSIONS
  These proteins, therefore, are potential muscular dystrophy biomarkers for monitoring disease progression and therapeutic response in both preclinical and clinical studies.
authors: Worldwide Research & Development, Pfizer Inc., Groton, CT, USA.
authors: Peter M
authors: PM
authors: Burch
authors: John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, UK.
authors: Oksana
authors: O
authors: Pogoryelova
authors: Worldwide Research & Development, Pfizer Inc., Groton, CT, USA.
authors: Richard
authors: R
authors: Goldstein
authors: Worldwide Research & Development, Pfizer Inc., Cambridge, MA, USA.
authors: Donald
authors: D
authors: Bennett
authors: John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, UK.
authors: Michela
authors: M
authors: Guglieri
authors: John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, UK.
authors: Volker
authors: V
authors: Straub
authors: John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, UK.
authors: Kate
authors: K
authors: Bushby
authors: John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, UK.
authors: Hanns
authors: H
authors: Lochmüller
authors: Worldwide Research & Development, Pfizer Inc., Cambridge, MA, USA.
authors: Carl
authors: C
authors: Morris
keywords: Muscular dystrophy; biomarker; creatine kinase; fatty acid binding protein 3; myosin light chain 3; skeletal troponin I