Lottery win helps baby's treatment for rare disease

Fourteen-month-old infant - Fatima, who was afflicted with a killer muscular disorder, underwent a Rs 16 crore revolutionary gene therapy — Zolgensma, at a Bengaluru hospital after winning a lottery.

Bengaluru (Karnataka): Parents of babies with Spinal Muscular Atrophy (SMA), a rare genetic disease, came together at an event organised by Bangalore Baptist Hospital in the city on Tuesday.

Fourteen-month-old infant 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 Baptist Hospital of Bengaluru.

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. The cost of this medicine is about 2.1 million US dollars, which is roughly about Rs. 16 crore.

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 signalling is coded by a gene for which everyone has two copies — one from the mother and the other from the father.

There is gradual improvement. She is now able to move her leg. It will take time to become like a normal child.

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).

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