Anonymous donor gives S$1.5 million to baby with rare disorder in S'pore for treatment
In August 2022, the family of Zayn Bin Nabeel Abdat, an infant diagnosed with Spinal Muscular Atrophy (SMA) Type 1, launched a campaign to raise money for a S$3 million treatment.Without intervention, affected children may die before the age of two because of progressively weakening respiratory muscles and respiratory failure, according to the National Organisation of Rare Diseases (NORD).
The crowdfunding campaign hit S$1.5 million with the help of family, friends and the public, before donations gradually slowed.
However, a very generous anonymous donor then stepped in and topped up the shortfall to meet the S$3 million target, ensuring the family raised enough for the treatment.
S$3 million raised
On the crowdfunding platform Ray of Hope, it was revealed that the goal was to raise S$3,015,167.50 for the now five-month-old Zayn's treatment.
Even though the site showed that donations were only halfway there, the family met its target with the help of the anonymous donor, former Member of Parliament, Amrin Amin, wrote in a Facebook post on Nov. 30.
An update on the site stated they achieved their target ahead of schedule with "several large anonymous donations" and closed the campaign at the end of November.
Amrin wrote:
"This is very touching. Such kindness reminds us there’s still so much compassion and positivity in our community, even during this difficult time," Amrin shared.
Treatment for rare disorder
According to the NORD, SMA is a genetic neuromuscular disease that results in worsening muscle weakness and poor muscle tone over time, without appropriate treatment.
This can affect one's ability to swallow, suck, and breathe. It happens to approximately one in 10,000 live births.
While there is no known cure for SMA, there are currently three available treatments: Zolgensma, Risdiplam, and Spinraza.
According to Zayn's family, the infant was prescribed Risdiplam and Zolgensma, the latter being a "one-time gene therapy" often used for children under two years old.
The treatment involves the replacement of the dysfunctional SMN1 gene, which encodes a protein important to maintaining one's motor neurons.
However, the treatment, costing about S$3 million, is only available in Singapore via the Special Access Route, according to The Straits Times.
On Aug. 18, Zayn's family said they decided to order Risdiplam first, an oral drug that is administered daily, due to the "urgency of Zayn's condition".
Each bottle of Risdiplam costs more than S$15,000 and lasts him 64 days.
He started on the Risdiplam treatment in August and has since stopped.
In a post on Nov. 23, Zayn had stopped his Risdiplam treatment for a week and was "cleansing" his body in preparation for his Zolgensma treatment.
Going for Zolgensma treatment
On Dec. 1, the Facebook page Walk With Zayn announced that Zayn has undergone his treatment.
After the procedure, which lasted for an hour, the post, written from Zayn's perspective, said Zolgensma "has now been infused into my vein, a new gene is now flowing and circulating through my body".
However, this is not the end of the family's challenges.
The family shared with ST that Zayn may also have to continue taking Risdiplam daily.
Also, Novartis, the pharmaceutical company which manufactures Zolgensma, announced in August that acute liver failure is a known side effect, following two patient fatalities.
As such, Zayn may also have to take steroids to treat this.
You can find more information about SMA and follow Zayn's journey on the Facebook page Walk With Zayn.
https://mothership.sg/2022/12/anonymous-donor-baby-rare-disorder-singapore/
