A 14-month old girl faced a bleak future afflicted with a killer muscular disorder, but a Rs. 16 crore 'revolutionary' gene therapy she underwent at a city hospital after winning a 'lottery' has given her a new lease of life.

The patient, daughter of parents from Bhatkal town in the coastal Uttara Kannada district in Karnataka, is recovering after she was given 'Zolgensma', the gene therapy at Bangalore Baptist Hospital late last month. She emerged "a lucky winner of a lottery" through a compassionate access programme by drug major Novartis that helped her get the costly treatment, affordable only by multi-millionaires, the hospital said. "The cost of this medicine is about 2.1 million US dollars, which is roughly about Rs. 16 crore," hospital Director (CEO) Naveen Thomas said. "There is gradual improvement. She is now able to move her leg. It will take time to become like a normal child," her father told PTI.

The toddler was diagnosed with Spinal Muscular Atrophy or SMA, a disease caused by loss of nerve cells, which carry electrical signals from the brain to the muscles. The protein needed for this signaling is coded by a gene for which everyone has two copies --- one from the mother and the other from the father, according to Thomas. He said a child develops this disorder only if both the copies were faulty and without treatment, this disease was ultimately fatal. But the problem is that the treatment is out of reach of most people.

"Only multi-millionaires can afford it! Current treatment options range from medicines, which increase these proteins to replacing the faulty gene. Zolgensma, a gene therapy is a revolutionary treatment, which aims at curing the disease by replacing the faulty gene", he said. "For the first time in Karnataka, Zolgensma was given at Bangalore Baptist hospital to a child who was the lucky winner of a lottery through a compassionate access programme by Novartis", Thomas said.

Incidentally, the couple had earlier lost a child, who was also suffering from SMA. "On the 21st day of the 21st year of the 21st century, the baby was given the injection, which is a one-shot cure for this rare disease, said Dr Ann Agnes Mathew, Consultant Paediatric Neurologist and Neuromuscular Specialist. At present there were about 200 children getting treatment in the Baptist Hospital which is specialised in genetic diseases, more specifically SMA and Duchenne muscular dystrophy (DMD), said the doctor. She added that previous year alone, 38 children who were getting treatment in the hospital breathed their last in the absence of this expensive treatment. In this case, Thomas said: It is a dream come true for doctors in this field. We hope more children receive this treatment and many such treatments will become affordable in the future."