3-Year-Old Hyderabad Boy With Rare Disease Needed Rs 16 Crore Injection. How People Helped
Thousands of people came together online to save the life of a three-year-old boy in Hyderabad, Telangana, by helping him get an injection worth Rs 16 crore to treat a rare genetic disorder.
Ayaansh Gupta received the world’s most expensive drug Zolgensma, a single-dose intravenous injection, on Wednesday, at the Rainbow Hospital in Hyderabad after 62,450 people contributed Rs 14.84 crore through crowd-funding.
The boy’s parents, Yogesh Gupta and Rupal Gupta, had been waiting for more than two years to treat him after he was diagnosed with a progressive neuromuscular disease called Spinal Muscular Atrophy.
They were able to raise most of the money for his treatment through the crowd-funding platform ImpactGuru in around four months.
Mr Gupta thanked donors and doctors for their help. “Thank you so much… thanks to around 65,000 donors who came forward to donate and saved Ayaansh. We are very happy that we finally got this medicine for which we were waiting since a long time. This can change the life of Ayaansh… so we are very, very happy,” he said.
Mr Gupta said he and his wife were heartbroken when, after a few months of being born, their son was diagnosed with the rare genetic disease that made his hands and legs weak and he was not able to stand or sit without help.
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.
Ayaansh’s parents initially put him on the waiting list of pharma companies that sponsor the treatment of children with the rare disease. However, they failed to get a breakthrough after which they turned to online crowd-funding.
Dr Ramesh Konanki, who administered the gene therapy to the boy, said this would arrest the disease and Ayaansh could hope to achieve new growth milestones from now on.
“There are 800-900 people living with this (disorder). Three to four times that number of children with the disease die by the age of two. For Ayaansh, we managed to get crowdfunding in about four months’ time, and we are very happy,” he said.
The Rainbow Hospital has earlier given the Zolgensma gene therapy to two more children — in August, 2020, and April this year. In both those cases, US-based Novartis had provided the injection for free on compassionate grounds. The two children are said to be recovering well and making progress in physical growth and development, Dr Konanki said.
“Our Spinal Muscular Atrophy fundraisers have made history in India’s medical crowdfunding industry. We hope more families come forward to utilise our healthcare financing platform in their time of need to get the best available treatment for critical illnesses,” said co-founder of ImpactGuru Piyush Jain.
Last month, ImpactGuru helped raise a similar amount to help get the Zolgensma drug for a five-month-old child in Mumbai, Maharashtra, with the same disease.
Developed by the US biotechnology startup AveXis, which was acquired by pharmaceutical giant Novartis, Zolgensma was cleared for use in the US in 2019 and in UK this year.
According to the UK’s National Health Service, the treatment is given as a single intravenous infusion and contains a replica of the missing gene SMN1 that can enable mobility in babies and young children suffering from the rare genetic condition.