Toddler with rare genetic condition can be treated after $2m raised, drug registered
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Mr Achintha Pilapitiya, his wife Dulanjali Wakwella and their son Shamel, who suffers from Type 2 spinal muscular atrophy.
PHOTO: TAMIL MURASU FILE
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SINGAPORE - The couple were running out of time and options to raise funds to treat their 21-month-old with a rare genetic disorder, when, serendipitously, the drug was registered in Singapore.
It meant that the price of Zolgensma, a one-time gene therapy by pharmaceutical company Novartis, was reduced from $3 million to $2,397,300, and their insurer could help cover some of its cost.
Mr Achintha Pilapitiya and his wife, Ms Dulanjali Wakwella, both 33, now have sufficient funds to get the drug for their son, Shamel, who has Type 2 spinal muscular atrophy (SMA).
They raised $2.02 million in a crowdfunding campaign on Ray of Hope
“Everything happened when the campaign slowed down, and we were wondering what other ways there were to raise the money. We exhausted our resources and energies. So when we were informed by the hospital, it was the best news,” said Mr Achintha, a software engineer.
His wife added: “It’s the biggest dream come true and a huge load off our shoulders. Miraculously, the drug got registered so we want to thank the authorities and the drug company for getting it registered.”
The parents also expressed their gratitude to the parents of other children with SMA in Singapore who provided them with guidance, as well as donors who contributed to their now-closed crowdfund.
According to the Health Sciences Authority register, Zolgensma was approved on April 21.
The gene therapy, which was, until last November, known as the most expensive drug in the world, must be administered before the child turns two.
Shamel’s parents told ST on Friday that they are hoping to order Zolgensma through the National University Hospital in the coming weeks, and are hoping that their child will be able to get the drug in June.
If left untreated, SMA can cause a child’s motor function to decline with age. Problems include respiratory insufficiency, swallowing dysfunction, motor contractures and fractures.
In an update on the crowdfunding page, Ray of Hope said that with the funds raised outside of its platform by Shamel’s parents, the campaign target has been fulfilled, and it will proceed to transfer the funds to the hospital.
“We’re grateful for the community’s support and very glad that Baby Shamel can now receive this life-changing treatment. We will continue to keep donors updated on Baby Shamel’s treatment,” it added.
