Al Jalila Children’s Speciality Hospital, the only hospital specialised in paediatric medicine in the UAE, today announced plans to introduce spinal muscular atrophy (SMA) gene therapy for the first time in the UAE.
The therapy, Zolgensma (AVXS-101), will be administered to a five-month old girl who suffers from SMA, a genetic disease caused by a mutation in the survival motor neuron 1 (SMN1) gene which leads to debilitating and often fatal muscle weakness. In the treatment provided for the first time in the UAE, the young girl will be receiving the full gene therapy service under one roof, starting from clinical identification and genetic diagnosis, to the soon to be infused SMN1 gene therapy and subsequent follow up and management. The therapy is approved by the US Food and Drug Administration and health authorities in the UAE.
Dr. Mohammed Al-Awadhi, COO of Al Jalila Children’s, said “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. For the first time in the UAE, a patient will be receiving the full service of gene therapy under one roof. Experts from Al Jalila Children’s Neurology Center of Excellence and Genomics Center and other supporting departments will be adopting a 360-degree approach in providing this innovative treatment that addresses the root cause of the young girl’s disease and ultimately changing her life and giving her hope for the future.”
“The fact that the cost of the treatment was covered by an insurance company highlights the integrated healthcare system offered by Dubai that facilitates wide access to quality specialised care,” added Al-Awadhi.
The young patient was diagnosed within 48 hours in a genetic test conducted by Al Jalila Children’s Genomic Center. The test usually has a turnaround time of 2-4 weeks if it is done abroad.
SMA is a genetic disease caused by a mutation in the survival motor neuron 1 (SMN1) gene. The gene encodes the survival motor neuron (SMN) protein – a protein found throughout the body, which is critical for the maintenance and function of specialised nerve cells, called motor neurons. Motor neurons in the brain and spinal cord control muscle movement throughout the body. If there is not enough functional SMN protein, the motor neurons die, leading to debilitating and often fatal muscle weakness.
