SINGAPORE — A couple’s desperate attempt to save their 22-month-old son’s life by appealing to the public for funds has been successful with the family now able to pay for a one-time-only gene therapy treatment costing $2.868 million.
The sum was raised over 10 days via more than 29,000 donors.
“We are completely blown away by the level of support, love and generosity that poured in,” said Devdan’s parents Dave Devaraj and Shu Wen, both 33, on the Ray of Hope charity website.
“We are deeply grateful. You have given Devdan a second chance at life,” they added.
The toddler will receive the treatment soon at the National University Hospital (NUH).
Devaraj and Shu launched their crowdfunding campaign on 3 August and met their target amount last Friday.
Spinal Muscular Atrophy
Devdan, who turns two in October, was born with Type 2 Spinal Muscular Atrophy (SMA) – a rare condition that damages nerve cells in the brain and spinal cord. SMA leads to progressive muscular weakness and trouble with activities such as breathing, speaking, swallowing, and walking.
Zolgensma, which is the world’s costliest drug, is a gene therapy treatment for children under two years with SMA. It was approved by the US Food and Drug Administration in 2019 and will have to be imported into Singapore under the Special Access Route as it is not yet approved by Singapore’s Health Sciences Authority.
Zolgensma is designed to target the genetic root cause of SMA by replacing the function of the missing or non-working survival motor neuron 1 (SMN1) gene. This will, in turn, stop the progression of SMA and sustain the remaining muscle function needed for children to survive.
Should Devdan miss this window to receive the one-time treatment, the remaining options are daily oral medication and intrathecal injection every 4 months for the rest of his life. Patients on these alternative therapies will have to be on them for life.
When contacted, an NUH spokesperson said it has seen about three new patients with SMA every year over the past three years.
“If left untreated, motor function is expected to decline with age, with attendant problems of respiratory insufficiency, swallowing dysfunction, motor contractures and scoliosis, hip dislocation, osteoporosis and fractures,” the spokesperson explained.
Devdan is currently receiving multidisciplinary supportive care at NUH and the hospital is unable to comment further due to patient confidentiality, the spokesperson added.
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