DUBAI, The mobility of an Egyptian toddler who suffers from a rare genetic condition that causes muscle weakness and can affect a child’s ability to develop is showing improvement following gene therapy treatment at Al Jalila Children’s Specialty Hospital.
Spinal muscular atrophy (SMA) affects up to only one in 10,000 children and can limit the ability to crawl, walk, sit up, and control head movements. Al Jalila Children’s offers one of the largest gene treatment programme for SMA in the world and by far the largest programme in the UAE.
Rasheed first visited the hospital 12 months ago ahead of his first birthday and has since undergone a Zolgensma injection. When his clinical team met earlier this month for one-year post-therapy physical and occupational assessments, they found he was demonstrating improved upper limb movement.
“We are pleased with Rasheed’s progress,” said Dr. Haitham El Bashir, Paediatric Neurorehabilitation Consultant and Head of Neurosciences Centre of Excellence at Al Jalila Children’s Specialty Hospital. “He can now move his shoulders against gravity while in a sitting position, he can transit from lying to sitting with minimal assistance from his hips, he can stay kneeling for at least five minutes, and he has started to shuffle his way around on his bottom.”
With August marking SMA Awareness Month, Dr. El Bashir urged attention among parents and outlined the early symptoms as general floppiness, problems with breathing and swallowing, and continual signs of weakness.
“In our experience, the sooner you identify the condition and start the treatment, the better outcome you can achieve,” he said. “Some children can lose some skills they have gained because they become weaker with time, and there is a huge risk because the rapidity of muscle deterioration is such that it can affect breathing and swallowing, which can threaten the child’s life.
“Early identification and treatment are beyond doubt significant to the child’s wellbeing because a patient treated at two weeks of age will do better than one treated at the age of two. That is why it is especially important to have newborn screening. SMA is easy to diagnose through a simple 24-hour genetic test.”
In the two years since Al Jalila Children’s launched its dedicated gene therapy, the hospital has provided the Zolgensma treatment to 41 patients from 13 countries from across Europe and the Middle East, with the largest number of patients arriving from Turkey.
Source: Emirates News Agency