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An advanced gene replacement therapy has helped save the lives of a pair of seven-month-old Emirati twins in Abu Dhabi.
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The babies were diagnosed with spinal muscular dystrophy (SMA1), a rare and debilitating genetic disorder that affects the nerve cells that control voluntary muscles. It weakens and wastes away the muscles of the body, affecting basic functions such as breathing and swallowing and progressing into paralysis.
After making a neurological assessment of the babies, Dr Hussein Matlik, consultant pediatrician and child neurologist at Burjeel Hospital in Abu Dhabi, said: “The first consultation with the babies showed that they were severely weak, had hypotonia, as well as areflexia, which is the absence of neurologic reflexes. This led us to do a genetic test and, as a result, the SMA diagnosis test came back positive for both babies.”
Dr Matlik explained that the symptoms of this devastating disease start manifesting in children in the first six months of their lives. "If left untreated, the disease can lead to death or the need for permanent ventilation by the age of two in more than 90 per cent of the cases," he said.
“We knew that we needed to act fast and Burjeel Hospital made every arrangement for the transformational treatment to be carried out as soon as possible," he continued. "Based on the available data, we are confident about the success of the therapy.”
Under the supervision of Dr. Amro El-Saddik, VPS Healthcare excellence director and his team, the infants were subjected to gene replacement therapy known as AVXS-101 (onasemnogene abeparvovec).
“This potentially transformational therapy targets the root cause of the disease with a single dose that introduces a functional SMN1 gene to restore SMN protein expression. Available data for previous administration of the therapy is reassuring and shows that the treatment is both effective and safe,” Dr El-Saddik said about the one-time treatment.
Hopeful about the success of the treatment, the parents of the twins, Rashid Al Hasani and Mona Ali, said: “We are incredibly grateful to Dr Matlik and the team at Burjeel Hospital for diagnosing our children so swiftly and offering this life-saving gene therapy treatment. We are feeling very positive, and we hope Allah is listening to our prayers and will shower his blessings upon our children and our family.”
saman@khaleejtimes.com
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