But we can help some. Thankfully, there are rays of light in this darkness.
Kamu* is from a remote village in West Bengal. He was 9 years old when he met the doctors at CMC. He does well at school and is well known for his sense of humour among his friends.
He was normal at birth. But by two years old, his tummy was big (distended). Doctors at CMC were finally able to diagnose the problem.
A test of his bone marrow showed a rare disorder called “Gaucher Disease”. His enzyme and genetic reports confirmed this. Only about 1 in 100,000 people have this disease.
This came as a big shock to his parents. Enzyme Replacement Therapy (ERT) is the best treatment for this disease. BUT it would cost about Rs.35 lakh every year.
On top of that, he would need admission to hospital, for an infusion, every 2 weeks. Yet more out of pocket expenses for his parents. They are daily wage labourers and this amount would be impossible for them.
All their hope was gone. They would have to watch their child continue to suffer from this disease.
However, in this dark tunnel of despair, some light appeared. CMC’s Medical Genetics team is studying Gaucher disease. They are testing a new drug to treat it, and invited Kamu to take part. Thankfully, the drug helped him.
The trial is over but he can still take the new drug, and for him, it is free. So, there is no cost to his parents.
