Hyderabad child with rare disorder gets world's costliest medicine through crowd-funding

The generosity of nearly 65,000 people has helped a three-year-old boy in Hyderabad get a life saving drug, billed as the world's costliest at Rs 16 crore, for treating Spinal Muscular Atrophy (SMA), a rare genetic disease.

Zolgensma, labelled as the "world's most expensive drug" and is a single dose intravenous injection, was imported from the US and administered to Ayaansh Gupta on Wednesday at a private hospital.

The treatment has given a new lease of life to the child, who was diagnosed with SMA when he was just one year old.

"This is an extremely rare condition which causes muscle weakness...the child will be unable to move his hands or legs, sit, stand or even walk. In fact, eating food is also difficult," said the boy's father Yogesh Gupta, working in a private firm in Hyderabad.

“The condition is also life threatening and the doctors said that my child's life expectancy was three to four years. It was therefore important to treat him right on time", he told PTI.

At first, it seemed impossible for the parents to raise Rs 16 crore for one dose. But then the couple decided to do it through appeals on social media.

In February this year, they started a crowd funding campaign to save their son's life and family friends also extended their support.

In three months, they managed to raise enough money to pay for the treatment.

Many celebrities, including Virat Kohli, Anushka Sharma, Anil Kapoor, Ajay Devgn and several from the TV industry came forward to help the couple.

"Around 65,000 people donated. It was made possible on May 22," Gupta said.

A family friend reached out to an MP, who raised the issue of cost of the medicine in Parliament, following which the central government gave Rs six crore tax exemption.

With the medicine being administered,Gupta expressed hope that his child's condition would improve in the coming months.

The child has been discharged from hospital and follow-up treatment would continue, he said.

Yogesh Gupta thanked all the 65,000 donors who helped them mobilise funds for the expensive treatment.

Rainbow Children's Hospital,where Ayyansh Gupta was treated, said he was administered Zolgensma on June 9.

Spinal Muscular Atrophy is a progressive neuromuscular disease caused due to a defect in SMN1 gene. The affected children develop muscle weakness involving upper and lower limbs initially, but over time develop breathing difficulty and swallowing difficulty, the release said.

SMA affects one in 10,000 children generally and there are nearly 800 children suffering from SMA in the country currently, it said, adding thrice as many children die before they even reach their second birthday.

Zolgensma is a single dose intravenous injection gene therapy, in which the defective SMN1 gene is replaced through adenoviral vector, it said.

Ayaansh's ImpactGuru.com fundraiser raised Rs 14.84 crore, cumulatively contributed from over 62,450 donors.

"The single highest donation received on the crowd funding campaign was Rs 56 lakh and in USD, was $7,000," Piyush Jain, Co-Founder and CEO, ImpactGuru.com, said in the release.

He said this is their third successful fundraiser for a SMA Type 1 case in the last four months and hoped that more families would come forward to utilize their healthcare financing platform in their time of need to get the best available treatment for critical illnesses.