Minor boy suffering from rare Gaucher disease gets Rs 2L sponsorship

Aurangabad, Nov 8:

A ray of hope of living life confidently emerged in Muhammed Saad s/o Shaikh Javed (6 years) after a Delhi-based company has agreed to sponsor him Rs 2 lakh to conduct enzyme replacement therapy from November to December.

Saad is suffering from Gaucher disease and was detected with the rare disease a couple of years. The hopeful persuasion of his father and consistent effort by the Genetic Clinic at MGM Hospital got paid off and the company - Sanofi Genzyme agreed to sponsor the kid under India Charitable Access Programme (InCAP).

The assistant professor and in-charge of Genetic Clinic at MGM, Dr Suvarna Magar confirmed that the healthcare company has sponsored Rs 2 lakh for the patient and the treatment has started on November 3. " The patient requires lifelong therapy with specific enzyme glucocerebrosidase. He will be given two vials (injections) on a gap of 15 days. The cost of each vial is Rs 25,000. Hence eight doses will be administered to him in two months," said Dr Suvarna.

The cost of treatment is around Rs 6 lakh per anum for a patient of weight 10 kg. Now, the patient's guardian will have to register the name on National Health Portal (NHP) through KEM Hospital-Mumbai (it is a single registration centre for patients suffering from rare diseases in Maharashtra). Then the union Ministry of Health and Family Welfare (uMoHFW) reviews the applications (on the waiting list) reviewing the severity of patients and decide upon funding accordingly, stressed Dr Magar adding that private crowdfunding to raise money for the treatment is also okayed under the rare disease policy.

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Lokmat Times highlighted story of unfortunate kids

It may be noted that the AurangabadFirst (Lokmat Times) has published the story on two unfortunate kids suffering from rare diseases like Gaucher and Mucopolysaccharidosis (MPS-I) on January 21, 2021. The treatment is very costly. The parents during interaction claimed that the treatment is unaffordable for them. For the past two years since the detection of rare diseases, these minors are facing problems like enlargement of the spleen, liver, brittleness in bones, blockage in lungs, abnormality in the face, heart functioning, blood circulation, crippleness in body structure, seal in normal growth, etc.

The replacement of enzymes or intravenous medications during the whole life is the only treatment for these patients. The genetic defect had pushed these minors as well as their parents to undergo stress and trauma, but they have not lost their hopes.