UAE public raises Dh6.6m for 'miracle' drug for toddler who struggles to breathe

Muhib has spinal muscular atrophy and will take his Zolgensma shot in the next two weeks

Muhib suffers from spinal muscular atrophy. He will be given a drug that may enable him to overcome some of the condition's worst symptoms. Photo: International Charity Organisation
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UAE residents have raised Dh6.6million ($1.8 million) to transform the life of a 19-month-old child who struggles to breathe with a “miracle” drug.

The UAE's International Charity Organisation issued an appeal on Friday to help Muhib, who suffers from spinal muscular atrophy.

In just four days, it collected Dh4 million to help him — with millions more now pledged for further treatment.

Abdulhakeem Abdullah, 37, the boy's father, told The National: “I’m overwhelmed. I will be forever grateful to the UAE and its good people.”

I’m overwhelmed. I will be forever grateful to the UAE and its good people.
Abdulhakeem Abdullah, Muhib's father

Muhib is being treated at Al Jalila Children's Specialty Hospital in Dubai and will receive the Zolgensma injection in the next 10 to 14 days.

His condition affects motor neurons, making the muscles weaker and leaving the patient struggling to move and breathe. Infants with it may die before their second birthday.

The “miracle” drug is the most expensive in the world and costs Dh8 million ($2.1m) for a one-time shot.

The drug does not cure the condition but it significantly improves quality of life, in many cases allowing the child to breathe unassisted and sit up unaided.

After considering the financial situation of the Abdullah family, the hospital shared the cost, reducing the price to Dh6.6 million.

Mr Abdullah, an accountant on a salary of Dh6,000 ($1,600) a month, said it would have been impossible to pay a fraction of the cost. The family lives in a studio flat in Sharjah, which costs Dh10,000 a year to rent.

“We were desperate. We reached out to different charities and knocked on so many doors,” he said.

“Some charities promised to support with Dh2.6 million, but the International Charity organisation collected Dh4 million in just four days.

“Today, the charity called me saying the donation process was completed successfully.

“The doctors said Muhib should take the injection before he turns two and we just had a few months left.

“My wife and I are thrilled that in a few days, our son will get the treatment and live with the support of people in UAE and 'Eyal Zayed' [Zayed's Sons].”

Last week, the Ajman charity took up Muhib's case.

“We received a request from Muhib’s family to help them due to their financial condition. We studied the file and decided to raise Dh4 million,” said a charity spokesman.

“We receive social, humanitarian and health cases and study them before appealing for donations or providing aid.”

They posted a video of the child in the hospital with his mother at his bedside asking for help to pay his medical expenses.

“He needs a very expensive injection. He must take the injection as soon as possible, without it he won't survive. To all the people in the UAE, please help me save my son Muhib,” the mother said in the video as she broke down in tears.

Muhib's journey so far

Muhib was born in May 2021 and in January 2022 he was taken to Al Jalila hospital for pneumonia. He was admitted to the intensive care unit as his condition deteriorated.

In March 2022, he was put on a ventilator and his parents were told he suffered from spinal muscular atrophy disorder.

“He suffered from weakness in lower limbs with no head control. Muhib cannot play and do anything that children of his age do,” Mr Abdullah said.

“The money will be deposited in the hospital's account in a couple of days. I can’t wait for the day when my son fully recovers and plays with his older brother.”

What is spinal muscular atrophy?

The condition affects one in 10,000 children and can limit the ability to crawl, walk, sit up, and control head movements.

Children as young as two months can display signs of the most common type of spinal muscular atrophy. It is a hereditary disease caused by a missing or faulty gene that the body requires to make a protein essential for motor neuron cell survival.

Without sufficient levels of the protein the motor neurons — nerve cells in the brainstem and spinal cord that control activities such as speaking, walking, breathing, and swallowing — die, leading to muscle weakness and atrophy.

There are four types of the condition.

Type 1 is the most severe and many children with it do not live past the age of 2. Muhib has Type 1 disorder.

Children with Type 2 may sit without support, but cannot walk on their own.

Type 3 is a milder form of the disease and resembles muscular dystrophy. Children are usually able to walk with difficulty and some eventually need a wheelchair. They usually have a normal life expectancy.

Type 4 is very rare. It starts in young adulthood and results in mild motor impairment.

Treatment includes the spinal injection that Muhib will receive.

It is a one-time infusion that transfers the missing survival motor neuron gene directly into body cells through a virus vector — tools commonly used by molecular biologists to deliver genetic material into cells.

In 2019, the Food and Drug Administration in the US approved Zolgensma to treat spinal muscular atrophy in children who are younger than 2 years old. It is the first gene therapy to receive FDA approval for the treatment of SMA.

Other recommended treatments include a syrup given to the child for the rest of their life and Spinraza injections, with four to be given in the first two months of treatment and then every four months for the rest of the patient’s life.

Since Al Jalila Children’s launched its dedicated gene therapy in 2020, the hospital has provided the Zolgensma treatment to more than 40 patients from 13 countries from across Europe and the Middle East.

Paul Pogba visits Al Jalila Children’s Specialty Hospital — in pictures

Updated: January 31, 2023, 6:06 PM