• Enzyme replacement therapy (ERT)
• Gene therapy – Under Development
• Stem Cell Therapy- Under Development
ERT with a larger number of late-onset patients is promising.
Myozyme is the first the first Specific Treatment for Pompe Disease.
Myozyme ® (alglucosidase alfa) is a lysosomal glycogen-specific enzyme indicated for use in patients with Pompe disease (GAA deficiency).
Myozyme has been shown to improve ventilator-free survival in patients with infantile-onset Pompe disease as compared to an untreated historical control, whereas use of Myozyme in patients with other forms of Pompe disease has not been adequately studied to assure safety and efficacy.
Cost of Treatment
The dosage depends on the weight of the body and approximate cost of Myozome for 1 year is as below. This need to be given life long.
Weight of the Body Approx Cost
Upto 10 Kg 35 Lac/ Year
11 Kg – 20 Kg 75 Lac /Year
21 Kg – 30 Kg 1.05 Crore / Year
31 Kg – 40 Kg 1.40 Crore/Year
2) Supportive cost
Along with the ERT there is a need of supportive care cost for Pompe Patients on Monthly Basis Like Physiotherapy, Monthly Infusion cost, Monthly regular medicines. Cost towards treatment of other specialties, Cost of various routine lab test etc.
This cost is min of Rs 30000 to Rs 60000 Per month
3) One time cost
Based on the condition of the patient, Age at which the Pompe is diagnosed there is a requirement of onetime cost to the parents and are as below:
– If the Patient is on Ventilator dependent
– One time cost for Ventilator, UPS back up etc Rs 3 Lac
– Additional Monthly supportive cost – Rs 5000 per month
– Atomized Wheelchair- Rs 75000 to 2 lac
– If the patient develops a Antibody after ERT. Patient has to undergo Immune Separation Therapy and cost 3 Lac per month to 5 lac per month for a
period of 6 months to 1 Year
– Prenatal Diagnosis for Suspected or second pregnancy cost Rs 25000 to Rs 50000
-New Born screening for selected families- Rs 10000