Effect of cellular therapy in a case of limb girdle muscular dystrophy

Alok Sharma., Amruta Paranjape., Hemangi Sane., Nandini Gokulchandran., Dhanashree Sawant., Shruti Shirke., Vivek Nair., Sanket Inamdar and Prerna Badhe

Limb Girdle Muscular Dystrophy (LGMD) is a group of heterogeneous autosomal hereditary neuromuscular disorders resulting in progressive muscular weakness, predominantly in the proximal girdle muscles. Currently the management of LGMD is palliative and does not cure or alter the disease pathology. Hence there is a need of an intervention that may be able to alter the disease pathology. We present a case of a 41-year-old female of LGMD (Delta sarcoglycanopathy) who underwent two intrathecal and intramuscular transplantations of autologous bone marrow mononuclear cells (BMMNCs) along with standard rehabilitation at the interval of 7months. Musculoskeletal Magnetic resonance imaging (MRI-MSK) was conducted before both the transplantations. Over 13 months she showed improvements in functional outcome measures such as Bergs balance score, north star ambulatory assessment and six-minute walk test. Her functional independence measure score was unchanged for the 13 months since intervention. Per the natural course of the disease there is progressive muscle weakness. Maintenance of functional independence, improved muscle strength as well as six-minute walk distance and maintained MRI-MSK parameters over 13 months in this patient suggest that, cellular therapy along with neurorehabilitation may alter or halt the progression of the disease in LGMD.

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DOI: http://dx.doi.org/10.24327/23956429.ijcmpr20170244
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