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

Basic and applied myology

Nandini Gokulchandra¹  Prerna Badhe¹  Alok Sharma¹  Amruta Paranjape²  Jayanti Yadav²  Rishabh Sharan³  Hemangi Sane³  Pooja Kulkarni³ 

[1] Department of Medical Services and Clinical Research, NeuroGen Brain and Spine Institute, Mumbai;Department of Neuro-Rehabilitation, NeuroGen Brain and Spine Institute, Mumbai;Department of Research and Development, 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：农业科学（综合）

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】

CC BY