Nineteen-month-old Laveen Jabbar Al Kutyashi. Image Credit: Supplied

Dubai: A 19-month old Iraqi girl has received a second chance at life, thanks to the generous intervention of His Highness Sheikh Mohammed bin Rashid Al Maktoum, Vice-President and Prime Minister of the UAE and Ruler of Dubai.

World’s most expensive gene therapy

The compassionate gesture will provide the little girl a complete reversal of her genetic condition with what is the world’s most expensive gene therapy worth Dh8 million.

The toddler, Laveen Jabbar Al Kutyashi, was diagnosed with Stage II Spinal Muscular Dystrophy (SMA), a genetic condition that delays motor development. Her condition would have progressively deteriorated without this one–time life-saving injection that can cure her SMA and reverse the symptoms so far.

Mother in desperate appeal

The parents of the little child cannot thank Sheikh Mohammed enough for covering the cost of treatment of their beloved daughter. Shaikh Mohammad was moved by the plight of the child when he watched a video posted by Laveen’s mother on social media appealing for help.

Dr Mohammad Al Awadhi, CEO of Al Jalila Children’s Speciality Hospital, told Gulf News, “Luckily for the child, when the family came here from Iraq as directed by His Highness Sheikh Mohammed, she was diagnosed at our centre for excellence for genome studies to be suffering from this genetic condition. Al Jalila Children’s Speciality Hospital is the first and only one hospital in the entire region to provide this remarkable gene therapy for SMA.”

Good prognosis

Dr Awadhi further continued, “The gene therapy treatment for this condition has only been approved by the US FDA three years ago and is actually a one- time injection. The treatment will take place in a few days. The child will be administered an infusion of the drug Zolgensma (AVXS-101) according to her body weight. The earlier this drug is given, the better it is as it stops the muscular degeneration. In this case, the little girl was recently diagnosed, so the prognosis for her is very good.”

Once the drug is administered, the child will be monitored by paediatiricians and will also be put through a rehabilitation programme. The doctors attending on her are hopeful of a complete recovery.

'Faith in humanity restored'

Parents of Laveen, Ibrahim Jabbar Mohammad and Masar Mondal, feel their faith in humanity has been restored by this magnanimous gesture of His Highness Sheikh Mohmmed. “When I got a call from Al Jalila Hospital, my wife and I couldn’t believe that the Ruler of Dubai would make such a gesture to us. You cannot imagine our happiness and the hope that this has given us. Without this, we would have lost our baby.”

Mohammad, 35, a sales executive in Iraq and his wife, Mondal, also 35, are parents to another six-year-old daughter.  “When Laveen was born, she was completely fine and had normal movements. However, by the time she was six months, her movements had stopped and we were worried. Doctors in Iraq suspected Stage II SMA. However, it was diagnosed only in October 2019 when we were able to travel to Turkey after the lockdown for the pandemic was lifted. Genetic tests carried out there confirmed our worst fears and we were in great despair. My wife made that desperate appeal for help on social media and imagine our surprise, when such a powerful leader actually made this gesture to help us. My daughter has a new lease of life, thanks to the generosity of Sheikh Mohmmed.”

What is SMA?

Spinal muscular atrophy (SMA) is a genetic disease affecting the central nervous system, peripheral nervous system and voluntary muscle movement (skeletal muscle).

Most of the nerve cells that control muscles are located in the spinal cord, which accounts for the word spinal in the name of the disease. SMA is muscular because its primary effect is on muscles, which do not receive signals from these nerve cells. Atrophy is the medical term for getting smaller, which is what generally, happens to muscles when nerve cells do not stimulate them.

SMA involves the loss of nerve cells called motor neurons in the spinal cord and the condition falls under the category of a motor neuron disease.