This case report describes the treatment of a 7-year-old boy diagnosed with Duchenne muscular dystrophy (DMD) using stem cell therapy. DMD is a genetic disorder causing progressive muscle degeneration. The boy showed symptoms of muscle weakness and difficulty walking from age 2. After receiving no treatment elsewhere, stem cell therapy was started in October 2012. Progress notes since then show improvements in arm flexion, reduced skin infection, and weight gain. The conclusion is that stem cell treatment for DMD shows clinically improved results, and better outcomes require early and ongoing multi-disciplinary care.

1. ( CASE REPORT )
Ratna Juwita
Solo 19 Jan 2013

2. 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

3.  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

6. SYMPTOMS
 Fatigue
 Muscle weakness & wasting
 Frequent falls
 Difficulty in walking
 Limitation of movement
 Difficulty in getting up from a lying or
sitting position

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

8. THERAPY
 Steroid
 Physical therapy
 Orthopaedic appliances
 Breathing aids
 Gene therapy
 Stem cell therapy

9. Potential uses of stem cells

11. 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

12. 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

13. PROGRESS NOTES

14. PROGRESS NOTES

15. 3 yrs 5 yrs 7 yrs

16. 20/10/12

17. 24 / 12 / 12

18. 14/01/13

19. Arm flexion

20. Tinea of the chest

21. 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

22. MATUR NUWUN