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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