A three-year-old boy from Hyderabad in India suffering from a rare form of spinal muscular atrophy (SMA) has been treated with what is said to be the world’s most expensive drug after tens of thousands of donors responded to his parents’ crowdfunding plea to enable them to purchase it, The Hindustan Times reports.
Ayaansh Gupta was given Zolgensma, a gene therapy medication manufactured by Novartis-owned American biotech company AveXis that was especially imported from the US, after his parents, Yogesh and Rupal Gupta, began a fundraising campaign on social media on 4 February.
By 23 May, the Guptas had already raised INR16 crore (£1.5 million) from some 65,000 donors moved by the story of their son’s plight.
Celebrities including India cricket captain Virat Kohli and his actress wife Anushka Sharma were among those helping the family to reach their fundraising goal by donating generously.
Ayaansh was duly treated with the single-dose intravenous injection at Rainbow Children’s Hospital at Vikrampuri in Secunderabad on Wednesday and kept under close observation before being discharged.
“Except fever, which the doctors say is quite normal for a couple of days, he is absolutely fine,” his father told The Hindustan Times, proudly sporting a “My son has designer genes” T-shirt.
Doctors have advised the family to keep their son in quarantine for two months to avoid complications developing but pronounced the treatment a success.
“Since his immune system is weak, there is a possibility of him getting other infections. The doctors asked us not to allow any visitors,” Mr Gupta explained.
Known by the chemical name onasemnogene abeparvovec, the treatment works by creating a new copy of the gene that makes the human SMN protein, a deficiency of which causes SMA.
Late last month, five-month-old baby Arthur Morgan became the first British child to be treated with Zolgensma at the Evelina London Children's Hospital thanks to the NHS.