Seven-month-old twins diagnosed with rare SMA1 disease receive once in a lifetime gene therapy
Abu Dhabi, United Arab Emirates: VPS Healthcare’s Burjeel Hospital has successfully administered advanced gene replacement therapy, in an effort to save the lives of seven-month-old Emirati twins. With the gracious support of the government and leaders of the UAE, the therapy has been carried out on the twins by the Abu Dhabi based hospital who diagnosed them with a rare and most severe form of Spinal Muscular Atrophy – Type 1 (SMA1), a highly fatal disease.
After visiting Burjeel Hospital for a neurological assessment with Consultant Pediatrician and Child Neurologist, Dr. Hussein Matlik, the twin babies were diagnosed with the rare and devastating monogenetic neuromuscular disease, SMA1, which is also known as Werdnig Hoffman disease. SMA is caused by a defective or missing survival motor neuron (SMN1) gene. The genetic disorder affects nerve cells that control voluntary muscles of the body resulting in the rapid and irreversible loss of motor neurons, affecting all muscle functions, including breathing, swallowing and basic movement. It leads to progressive muscle weakness, paralysis, and when left untreated in its most severe form, death.
“The symptoms of this devastating disease start manifesting in children in the first six months of their lives and if left untreated, the disease can lead to death or the need for permanent ventilation by the age of two in more than 90% of cases. 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.” said Dr. Matlik.
He added: “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. Based on the available data, we are confident about the success of the therapy. This is a big achievement for the hospital, and also another success story for the UAE’s healthcare ecosystem.”
Under the supervision of Dr. Amro El-Saddik, VPS Healthcare Excellence Director and his team, the infants have been subjected to AVXS-101 (onasemnogene abeparvovec) gene replacement therapy. 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.
Parents of the twins, Rashid Al Hasani and Mona Ali said they are optimistic about the treatment. Adding: “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.”
Genpharm’s CEO, Karim Smaira commented: “Genpharm and Burjeel hospital share common values in ‘Putting Patients First’. It was greatly evident through their great efforts and their thorough follow up from diagnosing this rare and devastating disease to securing promptly the required authorizations to make the treatment administration possible”.
Since 2012, Burjeel Hospital has been providing world-class, specialized private healthcare to the growing Emirate of Abu Dhabi. The hospital is part of VPS Healthcare, one of the leading healthcare groups in the UAE.
About Burjeel Hospital, Abu Dhabi:
Burjeel Hospital is Abu Dhabi’s premier private healthcare facility providing world-class, specialized and superior healthcare complemented by a warm and personalized human touch to the growing population of the emirate of Abu Dhabi.
A unit of the VPS Healthcare, Burjeel Hospital opened for patients in April 2012 as a Tertiary hospital under the auspices of the Health Authority for Abu Dhabi. At the core of the organization is a team of exceptionally skilled and highly experienced health professionals, collaborating as multidisciplinary teams with Guests and their families as the main focus, to provide the best healthcare experience.
Burjeel Hospital received the prestigious Joint Commission International (JCI) accreditation in June 2013 – 15 months after starting operations. Currently, the hospital is affiliated with some of the world’s best healthcare institutions for its centers of excellence and is now becoming a renowned center of referral for Cardiovascular, Orthopedics, Ophthalmology, Pediatrics, Obstetrics and Gynaecology, Reproductive Medicine and many more specialties handling complex laparoscopic and minimally invasive surgeries.
The hospital has full-fledged pharmacy, laboratory, and radiology units to serve Guests at any time of the day. Burjeel also houses a restaurant and a coffee shop, serving the finest and healthiest cuisines from across the world. Combining all-round specialization and expertise with most advanced technology, Burjeel offers the best in diagnostic, curative, as well as preventive aspects of healthcare.
About VPS Health:
VPS Healthcare: Affordable, Accessible, Sustainable Healthcare
VPS Healthcare has played a key role in revolutionizing the entire private healthcare sector in the UAE. Since its inception in 2007, the group has grown to become one of the largest healthcare groups owning and managing 24 hospitals, over 125 medical centers and retail pharmacy network. LifePharama, UAE’s largest pharmaceutical production company, is also owned and managed by the group. It is the only similar facility in the Middle East approved by the U.S. Food and Drug Administration.
With an enormous workforce of 10,000 personnel from various nationalities across the world, VPS Healthcare was founded on the principle of delivering quality and accessible healthcare services to all the needy in the world.
Committed to the welfare of the nation and its people, VPS Healthcare and its management have played a crucial role in the advancement of the medical sector in the UAE. The group believes in continuous evolution and keeps upgrading by itself reckoning the advancement in the healthcare sector internationally. VPS Healthcare is also into medical education.
By providing comprehensive patient management at international quality standards across the MENA Region and beyond and to the entire strata of community, VPS Healthcare reflects a brand image of excellence in healthcare delivery system. VPS Healthcare is recognised as an industry partner by the World Economic Forum (WEF) and is also a member of the MENA Regional Partnership Community of WEF.