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SINGAPORE – Couple’s desperate attempt to save the life of their 22-month-old son by appealing to the public for funds has been successful, with the family now able to afford a one-time gene therapy treatment costing $ 2.868 million of dollars.
The sum was collected in 10 days via more than 29,000 donors.
“We are completely blown away by the level of support, love and generosity that has poured in,” Devdan’s parents, Dave Devaraj and Shu Wen, both 33, told the Ray of Hope charity website. .
“We are deeply grateful. You have given Devdan a second chance at life,” they added.
The toddler will soon receive treatment at the National University Hospital (NUH).
Devaraj and Shu launched their crowdfunding campaign on August 3 and hit their target amount last Friday.
Spinal muscular atrophy
Devdan, who will be two years old in October, was born with spinal muscular atrophy (SMA) type 2 – a rare disease that damages nerve cells in the brain and spinal cord. AS causes progressive muscle weakness and problems with activities such as breathing, speaking, swallowing, and walking.
Zolgensma, which is the most expensive drug in the world, is a gene therapy treatment for children under two years of age with AS. It was approved by the United States Food and Drug Administration in 2019 and will need to be imported to Singapore through the special access route as it is not yet approved by the Health Sciences Authority of Singapore.
Zolgensma is designed to target the genetic cause of ADS by replacing the function of the missing or non-functioning survival motor neuron 1 (SMN1) uncomfortable. This, in turn, will stop the progression of SMA and maintain the remaining muscle function necessary for the survival of children.
If Devdan misses this window to receive the single treatment, the remaining options are daily oral medication and intrathecal injection every 4 months for the rest of his life. Patients who take these alternative therapies will have to take them for life.
When contacted, a spokesperson for NUH said they have seen around three new patients with SMA each year for the past three years.
“If left untreated, motor function should decline with age, with concomitant problems of respiratory failure, swallowing dysfunction, motor contractures and scoliosis, hip dislocation, osteoporosis. and fractures, “said the spokesperson.
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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