Autologous bone marrow mononuclear cell transplantation in Duchenne muscular dystrophy - a case report.
METHODS
Male, 9 FINAL DIAGNOSIS: Duchenne muscular dystrophy Symptoms: Hyporeflexia • hypotonia • weaknes of lower limbs


METHODS
- Clinical Procedure: - Specialty: Neurology.


OBJECTIVE
Congenital defects/diseases.


BACKGROUND
Duchenne muscular dystrophy (DMD) is a fatal, genetic, progressive, degenerating muscle disorder. Current treatment options are palliative. Newer options of cellular therapy promise to alter the disease process. Preclinical studies have successfully tested myogenic, neurogenic potential and dystrophin expression of bone marrow mononuclear cells.


METHODS
We treated a 9-year-old boy suffering from DMD with serial autologous bone marrow mononuclear cell transplantations followed by multidisciplinary rehabilitation. Brooke-Vignos score was 10 and he was wheelchair-bound. Over 36 months, gradual progressive improvement was noticed in muscle strength, ambulation with assistive devices, fine motor movements, Brooke-Vignos score, and functional independence measure score. Nine months after the transplantation, electromyography findings showed development of new normal motor unit potentials of the vastus medialis muscle.


CONCLUSIONS
Magnetic resonance imaging scan of musculoskeletal systems showed no increase in fatty infiltration. This case report provides early investigative findings or the restorative effects of cellular therapy in DMD.
Department of Medical Services and Clinical Research, NeuroGen Brain and Spine Institute, Mumbai, India.
Alok
A
Sharma
Department of Research and Development, NeuroGen Brain and Spine Institute, Mumbai, India.
Hemangi
H
Sane
Department of Research and Development, NeuroGen Brain and Spine Institute, Mumbai, India.
Amruta
A
Paranjape
Department of Neuro-Rehabilitation, NeuroGen Brain and Spine Institute, Mumbai, India.
Khushboo
K
Bhagawanani
Department of Medical Services and Clinical Research, NeuroGen Brain and Spine Institute, Mumbai, India.
Nandini
N
Gokulchandran
Department of Medical Services and Clinical Research, NeuroGen Brain and Spine Institute, Mumbai, India.
Prerna
P
Badhe
Autologous Bone Marrow Mono Nuclear Cells; Duchenne – congenital; Electromyography; Muscular Dystrophy; Stem Cell Transplantation