The baby girl, who won the hearts of UAE residents after the Ruler of Dubai personally promised to sponsor the treatment needed to save her life, successfully received the required gene therapy on Thursday.
“It is a day that made us truly speechless, we cannot say or explain how we feel now, but it was not a dose of treatment — it was hope for all of us,” Iraqi national Ibrahim Al Jabar, the father of Baby Laveen, told Khaleej Times.
Laveen suffers from a rare and fatal genetic disease called spinal muscular atrophy (SMA). In February, Ibrahim and his wife Massar Mundhar posted an emotional video, pleading for help as they learnt that the one-time injection that would save their baby’s life was the ‘world’s most expensive therapy’. The drug, called Zolgensma (AVXS-101), cost Dh8 million.
When the video reached His Highness Sheikh Mohammed bin Rashid Al Maktoum, Vice-President and Prime Minister of the UAE and Ruler of Dubai, he promised to shoulder everything — a gesture that changed the family’s life.
“I always believe that Allah will never leave us alone, and it was right, because Allah made us get the gesture of Sheikh Mohammed bin Rashid,” Ibrahim said.
The baby’s rare genetic disease, SMA, is characterised by weakness and wasting in muscles used for movement. It is caused by a mutation in the survival motor neuron 1 gene, which leads to a debilitating and often fatal muscle weakness.
Baby Laveen stayed in the hospital for a day on Thursday and will just have to visit the hospital twice a week for three months for medical check-ups and physiotherapy — all of which will be covered by the help extended by Sheikh Mohammed.
With the treatment, Laveen got the chance to live a normal life like any other kid, and the entire family is looking forward to the days ahead. “I have no words to express my gratitude,” said Ibrahim, who is currently staying in the country as a visitor.
The world’s ‘most expensive drug’
Zolgensma is a prescription gene therapy used to treat children less than two years old with spinal muscular atrophy (SMA), a rare genetic disease characterised by weakness and wasting in the muscles used for movement.
Given as a one-time infusion into a vein, the drug is designed to target the genetic root cause of SMA, a mutation in the survival motor neuron 1 (SMN) gene. Zolgensma replaces the function of the missing or nonworking gene.
The new gene tells motor neuron cells to produce more SMN protein. Motor neuron cells need SMN protein to survive and support muscle functions.
“At approximately $2.1 million per patient, it is the most expensive treatment on the market. Zolgensma can be a one-time, life-saving treatment that allows for children with SMA to function in ways unimaginable just a few years ago,” according to a 2020 report on Forbes.
Al Jalila Children’s Speciality Hospital, the only hospital specialised in paediatric medicine in the UAE, first used the therapy in November 2020 on a five-month-old girl.
“We in Al Jalila Children’s are extremely proud that we are able to provide this gene therapy, an emerging revolutionary class of personalised treatments, where a defective gene — like the SMN1 gene in SMA patients —is injected back to patients to restore the gene and its biological function,” Dr Mohammed Al-Awadhi, COO of Al Jalila Children’s, has said in an earlier report.