Baby with rare disorder in need of lifesaving treatment

KUANTAN: One-year-old Muhammad Athif Fahri is in a race against time. He suffers from spinal muscular atrophy (SMA) Type 1 and desperately needs treatment, including a single dose of Zolgensma gene therapy, costing RM9.7 million that is administered via an intravenous infusion.

His 33-year-old parents, Nur Azizah Zamri, an intensive care unit (ICU) nurse at Hospital UIA Kuantan and Mohd Farhan Mohd Sait, a private sector clerk, are at their wits’ end, over the daunting task of raising the money for his treatment and have put out an urgent appeal to the public.

Nur Azizah told theSun that everything seemed normal at birth. But as they were about to be discharged from the hospital, doctors noticed an incomplete moro reflex, which is a lack of reflex action from Muhammad Athif when startled.

Concerned, doctors immediately placed Athif in the neonatal ICU. Blood samples were sent for testing and a month later, it was confirmed that he was suffering from SMA Type 1.

“I have been on unpaid leave since the end of last year to look after Muhammad Athif full-time. It has been a nightmare. With our combined salary of about RM6,000, we are unable to afford the treatment my son requires,” Nur Azizah said.

The couple, who hail from Taman Permatang Badak Maju, here, have two other children, aged six and five.

Nur Azizah said Muhammad Athif is currently receiving treatment at University Malaya Medical Centre in Kuala Lumpur, under the care of Prof Thong Meow Keong.

He needs to undergo Zolgensma gene therapy which is approved by the US Food and Drug Administration and the Health Ministry for the treatment of SMA in children under two years old.

“However, there are no subsidies for it, so patients have to fund their treatments themselves, which can be carried out in Malaysia.

“The hospital can import the drug and administer it in the country,” she said, adding that Zolgensma gene therapy is highly effective in improving motor function in SMA patients and is known to halt or even reverse the effects of the disease, providing hope for families like hers who are affected by this devastating condition.

“The treatment cost includes US$2.125 million (RM9.7 million) for the drug alone, which is administered as a single dose and is a one-time, intravenous infusion. The extra money is needed to cover ancillary medical costs.”

Children who have undergone treatment with Zolgensma can stand and even walk without assistance.

SMA News Today, a US portal specialising in the latest news and information about the treatment of SMA, states the following: “Large numbers of SMA patients given Zolgensma achieved at least one new motor milestone, which differs from the results seen in untreated SMA.

“Although motor milestones were achieved later than in children unaffected by SMA, an improvement was evident throughout the Zolgensma gene therapy. Overall, it was concluded that the available evidence supports Zolgensma as an effective treatment for SMA, especially for young patients with early disease.”

However, Zolgensma gene therapy comes with some side effects, such as an elevated risk of liver injury and low platelet count. The incidence of this is higher in infants who are older than eight months at the time of treatment.

“We have to start my son on the treatment before he turns two years old, so we have less than 12 months to raise the money. For now, his condition is being managed by taking an alternative drug called Risdiplam.

“This drug will help slow the progression of SMA in his body. It costs RM40,400 per bottle and each bottle lasts for 30 days. Muhammad Athif is currently on his fifth bottle, and thankfully, we have raised enough funds to purchase enough Risdiplam for the rest of the year.

“At the moment, Muhammad Athif can move his arms and hands. He wasn’t able to do this before taking Risdiplam. But still, he is breathing with the help of a non-invasive ventilator bipap machine,” she said.

Those who wish to contribute to Muhammad Athif’s treatment can deposit donations to Nur Azizah Zamri at Maybank account No. 153056155580.

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