Halting of functional decline in a case of duchenne muscular dystrophy after cellular therapy

Halting of functional decline in a case of duchenne muscular dystrophy after cellular therapy

Author: 
Dr. Alok Sharma, Dr. Prerna Badhe, Dr. Hemangi Sane, Suhasini Pai, Pooja Kulkarni, Dr. Khushboo Bhagwanani and Dr. Nandini Gokulchandran
Abstract: 

Duchenne muscular dystrophy (DMD) is an X-linked genetic myopathy characterized by progressive skeletal muscle degeneration and weakness. Recent studies have shown that stem cell derived exosomes promote angiogenic and cardioprotective function of cellular therapy. With no known cure, cellular therapy has shown some promise in altering the disease process. We report a case of DMD treated with autologous bone marrow mononuclear cell (BMMNC) transplantation followed by long term multidisciplinary rehabilitation. At follow up assessment of 4 and 13 months after cellular therapy, qualitative improvements like increased stamina, decreased calf muscle tightness, reduced toe walking, improved gait and balance were witnessed. Functional Independence Measure improved from 93 to 96. The North Star ambulatory score and Berg balance score were maintained and no functional deterioration were evident over 13 months post cellular therapy.This case report highlights the improvements in function as well as halting of progression of the disease over a period of 13 months after cellular therapy. No adverse events were observed. The improvements provide an evidence of the restorative and disease modifying benefits of cellular therapy in DMD. More randomized clinical studies will be needed to effectively establish the therapeutic benefits of cellular therapy in DMD.

Paper No: 
1170