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Effect of cellular therapy in progression of Becker’s muscular dystrophy: a case study

Alok Sharma, Hemangi Sane, Nandini Gokulchandra, Rishabh Sharan, Amruta Paranjape, Pooja Kulkarni, Jayanti Yadav, Prerna Badhe
  • Alok Sharma
    Department of Medical Services and Clinical Research, NeuroGen Brain and Spine Institute, Mumbai, India
  • Hemangi Sane
    Department of Research and Development, NeuroGen Brain and Spine Institute, Mumbai, India
  • Nandini Gokulchandra
    Department of Medical Services and Clinical Research, NeuroGen Brain and Spine Institute, Mumbai, India
  • Rishabh Sharan
    Department of Research and Development, NeuroGen Brain and Spine Institute, Mumbai, India
  • Amruta Paranjape
    Department of Neuro-Rehabilitation, NeuroGen Brain and Spine Institute, Mumbai, India
  • Pooja Kulkarni
    Department of Research and Development, NeuroGen Brain and Spine Institute, Mumbai, India | publications@neurogen.in
  • Jayanti Yadav
    Department of Neuro-Rehabilitation, NeuroGen Brain and Spine Institute, Mumbai, India
  • Prerna Badhe
    Department of Medical Services and Clinical Research, NeuroGen Brain and Spine Institute, Mumbai, India

Abstract

Becker muscular dystrophy (BMD) is an inherited disorder due to deletions of the dystrophin gene that leads to muscle weakness. Effects of bone marrow mononuclear cell (BMMNC) transplantation in Muscular Dystrophy have shown to be safe and beneficial. We treated a 20-year-old male suffering from BMD with autologous BMMNC transplantation followed by multidisciplinary rehabilitation. He presented with muscle weakness and had difficulty in performing his activities. The BMMNCs were transplanted via intrathecal and intramuscular routes. The effects were measured on clinical and functional changes. Over 9 months, gradual improvement was noticed in muscle strength, respiratory functions and North Star Ambulatory Assessment Scale. Functional Independence Measure, Berg Balance Score, Brooke and Vignos Scale remained stable indicating halting of the progression. The case report suggests that cellular therapy combined with rehabilitation may have possibility of repairing and regenerating muscle fibers and decreasing the rate of progression of BMD.

Keywords

Autologous bone marrow; Mononuclear cells; Becker muscular dystrophy; Manual muscle strength

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Submitted: 2015-09-17 08:22:28
Published: 2016-03-31 15:58:52
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Copyright (c) 2016 Alok Sharma, Hemangi Sane, Nandini Gokulchandra, Rishabh Sharan, Amruta Paranjape, Pooja Kulkarni, Jayanti Yadav, Prerna Badhe

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