Two families in the UAE whose young children are battling a devastating disorder are seeking support to raise the Dh8 million for life-saving treatment.
Hayat Mabda, 40, from Ajman, is facing a race against time to help her seven-month-old son Saif, who was recently diagnosed with spinal muscular atrophy.
The Syrian parents of 10-month-old Zain Mohamed Bazerbashi, in Abu Dhabi, are in the grips of their own fight to help their son overcome the debilitating condition.
Both have type 1, the most severe form of the rare genetic disease, which affects about one in 10,000 children and causes a rapid loss of motor neurones, leading to problems with breathing, swallowing and overall movement.
The most effective treatment, Zolgensma, is also the world’s most expensive drug, costing Dh8m for the one-time infusion.
Both families are exploring ways to secure the crucial funds.
'My child's life depends on treatment'
“Saif is my first baby following three miscarriages since 2017. When he was born I looked at him and felt like I held the world,” said Ms Mabda.
"My child's life depends on this medication. The doctors have said that if treatment is not given before Saif’s one-year birthday, his condition will deteriorate," the mother said.
Saif was diagnosed with SMA at six months old but his mother had spotted warning signs earlier.
“We went to Al Qasimi hospital in Sharjah after I noticed he wasn’t able to control his movements or hold his head up despite being around five months old,” she said.
Doctors in Sharjah and Dubai both confirmed he suffered from the degenerative disease.
They told Ms Mabda she must be careful when carrying or bathing him.
“I have to support his whole body to avoid any injuries since he can't control his movement,” she said.
Even when seated in his reclining chair, Saif’s mother has to support his head with her hands.
His mother had to leave her job at a beauty salon after giving birth to care for him. His father works at a private company.
“We can barely make ends meet with my husband’s salary. We can pay regular living costs but certainly not for his medication,” said Ms Mabda.
Nearly three weeks ago, Saif faced difficulty breathing and was taken to the emergency unit at Al Qasimi hospital.
“We were told he needs medical equipment to help him breath and to clean his chest from mucus. They cost around Dh17,000 we don’t have.”
Families try to maintain hope
Duha Almasri said her son Zain was diagnosed with the same life-threatening disease by doctors in Syria in June.
“When we returned, doctors in the UAE, confirmed the diagnosis,” she said.
“Doctors said the sooner he receives the treatment the better.”
His parents had to call an ambulance last month when Zain stopped breathing.
“He was taken to Tawam hospital where doctors are using a tube to help him breath since September 10,” said the stay-at-home mother.
Zain's father has been living in the UAE for 15 years and works at a fabric store.
The couple have two other children, aged five and three.
Both families have approached approved charities in the UAE to find out what assistance is available.
How does the treatment work and why is it so expensive?
Zolgensma is the only therapy designed to directly address the genetic root cause of the condition. It does this by replacing the function of the missing or nonworking gene, which is critical in making a protein essential to motor neurone cell survival.
It is extremely effective, but hugely expensive because its market is very small and it required a lot of research to develop, Dr Sushil Garg, a consultant neurologist at NMC Royal Hospital in Dubai, told The National.
“These therapies are extraordinarily expensive as it is first of its kind gene modifying therapy, approved for rare neuromuscular disorders,” said Dr Garg.
“Any medicine which is for gene modifying or work on [the] immune system has to undergo a lot of research and trials and approval processes, hence is pretty expensive.”