A 10-month-old in Bengaluru, Karnataka, is fighting for her life as she waits for a medicine that costs a whopping Rs 16 crore. Diagnosed with Spinal Muscular Atrophy, type 1, the child, Khyati, needs the medicine for gene therapy before she turns 2 if she is to get a fair chance for survival.
Khyati’s family is banking upon crowd-funding to help get the world’s most expensive drug, Zolgensma. Till now, they have been able to raise only Rs 3.3 crore.
Khyati’s mother, Jyoshna, said she is desperate for help to give her child a healthy life. “This is the age to play with toys. But my daughter is suffering from the last 6 months. She is with machines only. Please help us in her treatment,” she said.
The baby was diagnosed with the progressive neuromuscular disease after she struggled with simple tasks, considered development milestones, such as being able to lift her head at three months of age. Her parents consulted doctors and waited a month before DNA testing confirmed Spinal Muscular Atrophy when she was 5-months-old.
A person diagnosed with the disorder is unable to control the movement of muscles due to loss of nerve cells in the spinal cord and brain stem. It is treated with the help of gene therapy, which is expensive.
Raman, Khyati’s father, said: “Our little 10-month-old girl, Khyati, has been diagnosed with spinal muscular atrophy Type 1. It is a rare genetic disease and is 100 per cent fatal in children. She had difficulty in breathing and swallowing. And she was not able to crawl like any other child of the same age.”
“There is a treatment available, it costs 16 crores. Definitely we were not able to afford that kind of huge cost,” he added. “We started crowd-funding for her treatment and so far, with friends and family and relatives, we were able to raise close to 3.3 crores.”
An online crowd-funding process has also been started for Khyati on the Milaap website — milaap.org/khyati.
The officials at Bangalore Baptist hospital, which is handling Khyati’s case, said they have been helping 23 children with the same disease — seven of them have received the expensive gene therapy, including five in the last month, with medication free from Novartis.
Novartis follows a lottery system to provide the expensive medicines free-of-cost to people who need it. The hospital helps with the applications. Some children who failed to receive the therapy died due to the disease.
Last month, thousands of people had come together online to save the life of a three-year-old boy in Hyderabad, Telangana, by helping him get the injection through crowd-funding to treat a similar genetic disorder.