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

European Journal of Translational Myology

Nandini Gokulchandra³  Prerna Badhe³  Amruta Paranjape²  Rishabh Sharan¹  Hemangi Sane¹  Pooja Kulkarni¹  Alok Sharma³  Jayanti Yadav² 

[1] Department of Research and Development, NeuroGen Brain and Spine Institute, Mumbai;Department of Neuro-Rehabilitation, NeuroGen Brain and Spine Institute, Mumbai;Department of Medical Services and Clinical Research, NeuroGen Brain and Spine Institute, Mumbai

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

DOI  :  10.4081/ejtm.2016.5522

学科分类：骨科学

来源: Pagepress

【 摘 要 】

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.

【 授权许可】

Unknown