Parents 'race against time' to raise $3m to save baby with rare genetic disorder, Latest Singapore News - The New Paper

Parents 'race against time' to raise $3m to save baby with rare genetic disorder

This article is more than 12 months old

A few weeks after their first child was born, Mr Nabeel Salim Abdat and Ms Syahirah Yakub found that little Zayn's appetite was getting worse.

On Aug 4, Mr Nabeel took their baby to the National University Hospital (NUH), where Zayn fell seriously ill.

"While I was looking at some admission forms, I heard him cry," said Mr Nabeel, 30, who is due to start work as a data centre engineer in October. "About 30 seconds later, he was unconscious and he wasn't breathing."

Hospital staff managed to revive Zayn, who was later warded in NUH's intensive care unit.

His parents soon learnt that Zayn has a rare genetic disorder, and his best hope for survival may lie with a treatment that costs about $3 million, which they are now trying to raise.

On Aug 12, the seven-week-old boy was found to have Type 1 spinal muscular atrophy (SMA), a neuromuscular disorder that causes muscles to weaken. It can lead to declining motor functions a nd such issues as respiratory problems, scoliosis and osteoporosis, said an NUH spokesman.

If left untreated, the condition can lead to death by the age of two.

In response to queries, NUH said Zayn is currently receiving "multidisciplinary supportive care".

In the weeks before the diagnosis, he had been in and out of hospital due to fever and wheezing, the couple told reporters on Tuesday (Aug 23).

Ms Syahirah, a 31-year-old civil servant, said a paediatrician had also noticed that Zayn had poor reflexes and referred them to a specialist.

The couple said they were told Zayn's best hope is Zolgensma, a one-time gene therapy that is infused into a baby's veins to help stop the progression of SMA.

Zolgensma, approved by the United States Food and Drug Administration in 2019, is not authorised by Singapore's Health Sciences Authority, but it can be imported under the Special Access Route.

Costing almost $2.9 million, the treatment is often touted as the most expensive drug in the world.

"We thought, how are we supposed to get that amount of money?" said Ms Syahirah, describing it as an impossible task.

Last Friday, in a desperate attempt to achieve the "impossible", the couple started a crowdfunding campaign, Walk With Zayn, which has since raised more than $540,000 on charity platform Ray of Hope.

Ms Syahirah added that she has also appealed to the authorities to see if Zayn is eligible for the Rare Disease Fund, which provides long-term financial support to those who require high-cost medication for such conditions.

The Straits Times reported in January that a two-year-old boy with Type 2 SMA, a less severe form of the condition, was able to stand and walk with support after the public donated $2.87 million for his Zolgensma treatment.

Doctors have begun treating Zayn with Risdiplam, an oral medication taken daily. A bottle of Risdiplam, which lasts 64 days, costs more than $15,000.

SMA patients on Risdiplam or Spinraza, another drug that is injected into the spinal canal every four months, will have to be on them for life, NUH said.

The couple are hoping they can raise the money needed for Zolgensma, with Ms Syahirah noting that their son will still need therapy even after getting the drug.

Mr Nabeel said they are working on a deadline, as Zolgensma must be administered before the child turns two.

"We are racing against time and we are hoping that we can give him the medicine as soon as possible."