HYDERABAD: A three-year-old Hyderabad boy suffering from a rare genetic disorder was on Thursday administered a Rs 16-crore drug – said to be the world’s most expensive single-dose intravenous injection – as doctors attempted to cure his spinal muscular atrophy (SMA) type 1 condition that affects 1 in 8,000 children.
Ayaansh Gupta got a shot of the US-manufactured drug Zolgensma, thanks to over 62,400 people contributing Rs 14.84 crore towards his treatment through a crowdfunding site.
The remaining Rs 1.2 crore came from an international crowdfunding initiative and the boy’s family.
The Union government waived import duty of Rs 6 crore, without which the drug would have cost Rs 22 crore.
Children suffering from SMA type 1 rarely make it beyond two years of age without medical intervention.
“SMA is a progressive neuromuscular disease caused due to a defect in the SMN1 gene,” said Dr Ramesh Konanki, paediatric neurologist at Rainbow Children’s Hospital.
“Affected children show muscle weakness, involving upper and lower limbs initially, but over time develop breathing and swallowing difficulty,” added Dr Ramesh.
“Zolgensma is the single-dose intravenous injection of gene therapy, in which the defective SMN1 gene is replaced through adenoviral vector.
Ayaansh is doing fine and will be under observation for a day.” Ayaansh’s case had caught the attention of Bollywood celebrities and cricketers, with many of them donating to the fund and spreading the word.
“It is heartening to witness the power of crowdfunding.
A large number of donors came together to support Ayaansh Gupta’s family in their journey of saving their child.
The single highest donation received on the crowdfunding campaign is Rs 56 lakh and USD $7,000.
We are super ecstatic at the moment,” said Piyush Jain, co-founder and CEO, ImpactGuru.com.