Dubai, 11 November 2020: Al Jalila Children’s Speciality Hospital, the only hospital specialized in paediatric medicine in the United Arab Emirates, today announced its plan to introduce the first spinal muscular atrophy (SMA) gene therapy 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. 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 the concerned 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 personalized 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. Our Experts from Al Jalila Children’s Neurology Center of Excellence and Genomics Center along with other supporting departments will be providing a 360-degree approach for this innovative treatment addressing 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 that Dubai offers allowing increased access to quality specialized care” added Al-Awadhi.
The young patient was diagnosed within 48 hours after a genetic test was conducted by Al Jalila’s Children’s Genomic Center, a test which usually requires 2-4 weeks turnaround time if it is to be performed 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 specialized 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, then the motor neurons die, leading to debilitating and often fatal muscle weakness. SMA caused by mutations in the SMN1 gene.
About Al Jalila Children’s Specialty Hospital:
Al Jalila Children’s Specialty Hospital, the first and only paediatric hospital in UAE, caters for children and adolescents up to the age of 18 years. Inaugurated on November 1, 2016, Al Jalila Children’s Specialty Hospital is an ultramodern hospital with world-class teams of highly qualified healthcare experts that employs smart technology designed to enhance patient care and outcomes. The hospital also aims to foster clinical innovations, astute learning and development programs, and hosts cutting-edge research facilities. It is a 200-bed facility in a child and family friendly environment.
Find out more at www.aljalilachildrens.ae