Three-month-old baby with same disease that killed brother needs Dh8 million treatment

Spinal muscular atrophy causes rapid and irreversible loss of motor neurons that affect muscle functions

The parents of a three-month-old baby with a rare disease have been told she will die if she does not receive a drug that costs Dh8 million ($2.18m).

Mohammed and Nihal Al Alami live in Sharjah with baby Malak who has spinal muscular atrophy (SMA), a disorder that killed the couple's first child, Abdulrahman, in 2018.

The hereditary disease causes rapid and irreversible loss of motor neurons that affect muscle functions, such as breathing, swallowing, and overall movement.

It was really hard for us to hear this again, and to think we have to go through this experience again

The most effective treatment, Zolgensma, is also the world’s most expensive drug, costing Dh8 million for the one-time infusion.

Malak’s father, Mohammed Al Alami, 32, an accounts administrator, is already in debt from paying for his son’s treatment.

And, even if he "saved his whole life", he would not be able to afford Zolgensma.

He is afraid the couple will lose another child.

“She was really good when she was born,” said Mr Al Alami, a Palestinian born in the UAE,  talking about Malak.

“At seven days everything was normal, but I was thinking what if she had the same disease, what if she is sick? But I did not see anything to make me think she was.”

Mr Al Alami said he would seek constant reassurance from his family that she was healthy.

He consulted four doctors, but all dismissed his concerns.

“I used to go every two days to the last one. He was saying 'Mohammed, why are you coming daily? There is a huge chance to get infected with Covid-19'," he said.

“'You cannot keep coming to ask if she is normal or not. She is normal'."

Not convinced, Mr Al Alami took Malak to Al Jalila Children's Speciality Hospital.

The doctor ran some tests to see if she was suffering from the same disorder as her brother.

“After two days the results came,” Mr Al Alami said.

“She was positive. It was a shock for us. It was really hard for us to hear this again, and to think we have to go through this experience again.”

Although doctors have said her case has been helped by the fact it has been caught early, she still needs treatment.

Their first child had his SMA diagnosed after developing breathing problems at 27 days old.

The couple used up their Dh250,000 insurance limit within a month, but he was able to remain at the hospital for treatment for almost eight months for free as an emergency case.

Zolgensma was not an option at that time in the UAE, and he was too sick to travel to the US, so he was eventually discharged.

He died when he was almost 18 months.

“We were able to take good care of him, but then in November 2018, he passed away. He was one year and a half almost. It was very hard,” said Mr Al Alami.

SMA affects about one in 10,000 children. One in every 54 people carries the genetic defect that causes it and both parents must carry it to risk a 25 per cent chance of their child being born with the condition.

There are four forms of SMA.

Both of Mr Al Alami's children were born with SMA type 1, known as infantile spinal muscular atrophy or Werdnig-Hoffmann disease.

It typically presents after birth but before the age of six months, said Dr Sushil Garg, a consultant neurologist at NMC Royal Hospital in Dubai.

“Symptoms progress rapidly, and the majority of infants die before two years of age from respiratory failure," he said.

The drug she requires works by using harmless, genetically engineered viruses to increase protein levels. Once it is introduced, it travels around the body to help restore some of the protein missing as a result of the condition.

It is extremely effective, but hugely expensive because its market is very small and it required a lot of research to develop it, said Dr Garg.

“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.”

Follow baby Malak's updates on the Instagram account managed by her parents.