Fourteen-month-old Fatima, who was afflicted with a killer muscular disorder, is now blessed with a new lease of life after she underwent a Rs 16 crore 'revolutionary' gene therapy at a Bengaluru hospital after winning a 'lottery'.

Fatima, daughter of Mohammed Basil and Khadija from Bhatkal town in Uttara Kannada district of Karnataka, is recovering after she was given 'Zolgensma' -- the gene therapy -- at Bangalore Baptist Hospital late last month, news agency PTI reported.

She emerged "a lucky winner of a lottery" through a compassionate access programme by drug major Novartis that helped her get the treatment which is affordable only by multi-millionaires, PTI quoted hospital authorities as saying.

"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 Basil 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.

Thomas 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 Fatima's 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."

(With inputs from PTI)

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14-month-old lottery winner with killer disease gets new life after expensive therapy in Bengaluru - India Today

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