BENGALURU, JUNE 17
Three children suffering from the killer Spinal Muscular Atrophy (SMA) on Thursday received the Rs 16 crore drug each under the compassionate access programme by the drug major Norvartis, a doctor at the Baptist Hospital said.
Apart from them, three other children suffering from the same disease will get another drug, which costs about Rs 70 lakh each, Dr Ann Agnes Mathew of Baptist Hospital said.
“Under the lucky draw, these three children will get ”Zolgensma”, each of the doses costs Rs 16 crore,” Dr Mathew said.
According to her, two patients are from Hyderabad and one is from Bengaluru.
The children were chosen in a lucky draw, Dr Mathew said, adding this drug is effective for children below the age of two years.
Explaining about the disease, she said Spinal Muscular Atrophy or SMA is caused by loss of nerve cells, which carry electrical signals from the brain to the muscles.
The protein needed for this signalling is coded by a gene for which everyone has two copies – one from the mother and the other from the father, according to the doctor.
She said a child develops this disorder only if both the copies were faulty.
Without treatment, this disease was ultimately fatal, Dr Mathew said, adding the treatment is way too costly and out of reach of the common man and only a billionaire can afford it.
Dr Mathew said Zolgensma is a gene therapy, which aims at curing the disease by replacing the faulty gene.
“It’s a one-time shot and the child gets rid of it once and for all but it is effective for children below the age of two years,” she added.
Three other children suffering from the SMA who are above the age of two years will be given Spinraza, which costs Rs 70 lakh each, through an NGO, Dr Mathew said.
On February 16 this year, a 14-month-old girl Fatima from Bhatkal in Uttara Kannada district, received Zolgensma at the Baptist Hospital.
