CLINICAL IMPROVEMENT OFDUCHENNE MUSCULAR DYSTROPHY WITH STEM CELL THERAPY

( CASE REPORT )

Ratna JuwitaSolo 19 Jan 2013

INTRODUCTION

MUSCLE DYSTROPHY Are groups of inherited neuromuscular disorders Characterized by progressive weakness and

wasting of the muscles The most common form is Duchenne muscular

dystrophy

DUCHENNE MUSCULAR DYSTROPHY

1 : 3500 boys, X – linked disease Cause :

Mutation in the dystrophyn gene, located on the

x - chromosome

Part of the gene is missing

Body cannot make dystrophin

Progressive degeneration of all the muscles

SYMPTOMS

Fatigue Muscle weakness & wasting Frequent falls Difficulty in walking Limitation of movement Difficulty in getting up from a lying or

sitting position

DIAGNOSIS

Gower‘s sign Serum CK EMG Chromosome analysis Genetic test : dystrophin mutation Muscle biopsy

THERAPY

Steroid Physical therapy Orthopaedic appliances Breathing aids Gene therapy Stem cell therapy

Potential uses of stem cells

CASE

JAN 2010 Boy, 7,5 yrs, 19 Kgs Contact Dermatitis Muscle weakness, losing weight and develop a

difficulty in walking ( 2 yrs ) Referred to Dr. Soetomo Hospital Surabaya.

MAY 2012 Dr. Soetomo Hospital Surabaya :

Gowers’s sign : positive Serum CK : 109 ( normal < 171 ) EMG : myopathy Diagnosed : DMD NO TREATMENT

CASE OCT 2012

10 yrs, 16 Kgs 2 weeks : Tinea corporis on chest and neck Progressive muscle weakness Difficulty in walking, chewing and eating. Loss of appetite Falls frequently and joint stiffness

OCT 20, 2012 Stem cell therapy started

PROGRESS NOTES

PROGRESS NOTES

3 yrs 5 yrs 7 yrs

20/10/12

24 / 12 / 12

14/01/13

Arm flexion

Tinea of the chest

CONCLUSION

DMD treatment with stem cells shows clinically improved result

Treatment courses must be carried out from the early stage, with comprehensive multi dicipline approaches.

Longer period of observations is needed to obtain better result the treatment is still ongoing

MATUR NUWUN