Source: APO
- The 22-year-old Egyptian patient travelled to Dubai to receive the transformative one-time treatment at Medcare Royal Specialty Hospital for his genetic neuromuscular condition. This marks a significant advancement in gene therapy, as this patient is the second adult SMA patient worldwide to be treated with Novartis’ Itvisma as independently verified by the manufacturer Novartis.
- Medcare was the first private healthcare provider worldwide to administer the newly licensed Itvisma to a four-year-old international SMA patient last year, also confirmed by the manufacturer Novartis.
- The prevalence of SMA is estimated to be approximately 1 to 2 in 100,000 persons and DMD is estimated to be 1 in 3500 boys worldwide. Since 2020, Medcare has treated over 190 SMA patients and 20 DMD patients with revolutionary gene therapies.
Medcare Royal Speciality Hospital (MRSH) in Dubai has become the world’s first hospital outside the USA to offer a newly licensed intrathecal gene therapy to any adult patients with Spinal Muscular Atrophy (SMA). This one-time, life-altering treatment was recently administered to a 22-year-old Egyptian patient who had spent most of his life confined to a wheelchair, with few treatment options available. His case marks a decisive shift in the treatment landscape for SMA, a rare genetic disorder that progressively prevents patients from movement, independence, and, in severe cases, the ability to breathe.
Until now, gene therapy breakthroughs for SMA were largely limited to infants and young children below two years. Medcare is now opening the door for adult patients who were long excluded from cutting-edge medical care. SMA is a rare, inherited neuromuscular disorder that leads to progressive muscle weakness and loss of mobility, impacting movement, breathing, and swallowing. This is caused by a faulty or missing gene called SMN1, which is responsible for producing a protein essential for the survival of motor neurons.
Commenting on the advanced treatment, Dr. Shanila Laiju, Group CEO of Medcare Hospitals, said, “We believe that achieving the capabilities to provide SMA treatment to adults will rewrite the life stories of thousands of patients in the region and beyond. Being resourceful in this field shows how far we have come in the treatment of rare genetic diseases in this part of the world. At Medcare, our focus remains on bringing these life-changing innovations to patients who need them the most, regardless of the patient’s age or geography. Expanding access to advanced therapies for older patients is a meaningful step toward equity in care.”
“The procedure required a highly coordinated, multidisciplinary medical team approach because of the patient’s prior spinal fusion surgery for scoliosis, a frequent complication of SMA that significantly alters spinal anatomy and makes intrathecal access extremely difficult. Their expertise played a critical role in the successful delivery of the therapy. The introduction of Itvisma at Medcare builds on earlier milestones, including the hospital’s pioneering role in expanding gene therapy access to older children and patients beyond traditional eligibility limits as corroborated by Novartis, the medicine’s manufacturer. Globally, SMA affects approximately 1 in 10,000 live births and DMD affects 300,000 boys globally. Medcare has treated over 190 SMA and 20 DMD patients to date, welcoming individuals from across the Middle East, Europe, Asia, and Africa,” she added.
The medical team included Dr. Vivek Mundada, Clinical Lead of the Gene Therapy Unit at Medcare Hospitals in the UAE, alongside adult neurologist Dr. Sagar Kawale and anaesthesia specialists Dr. Neha Shahane and Dr. Ardalan Papari.
The patient, who was diagnosed with the rare SMA condition at just 18 months of age had been receiving four monthly intrathecal injections which could have been a life – long journey for him. The one-time gene therapy, Itvisma, works by delivering a functional copy of the missing SMN1 gene directly into the cerebrospinal fluid, addressing the root cause of the condition.
Following the successful administration of the therapy and a stable post-procedure outcome, Dr. Vivek Mundada remarked, “We could achieve this with the support of our highly specialised clinicians who followed a multidisciplinary approach, the visionary leadership of our organisation, and the UAE’s progressive healthcare environment that supports such advancements. The medical world considers this achievement as the next level of providing wider access to advanced gene therapy for adult SMA patients in our region.”
“For years, adults with SMA were told they had aged out of curative options. A one-time intrathecal gene therapy changes that conversation entirely. This is a life-changing treatment, and it opens new possibilities for many patients who were previously left without hope. What makes today especially meaningful is that our patient travelled from Egypt to Dubai specifically for this treatment. Dubai is fast becoming a genuine global destination for advanced gene and rare-disease therapy, and Medcare Royal Speciality Hospital is proud to be at the front of that movement,” he added.
Dr. Sagar Kawale said, “I am glad to be part of a team that delivered this breakthrough therapy. We are now able to offer meaningful treatment options to adult patients who were previously excluded. Beyond the complexities, this case is globally significant. This is a shift in how we approach SMA care, and it opens the door for many more patients to benefit from gene therapy. This development opens a multitude of doors for people who need this and helps establish new paths for the future of gene therapy.”
After undergoing the treatment, the patient remarked, “The experience of taking the Itvisma injection at Medcare was very positive. I was constantly well-informed, with the whole team taking very good care of me and being extremely responsive; this eased the process and made it smooth. The injection itself was also easy, with no pain felt around the injection area. I am just glad that I do not have to take multiple injections like I used to before. I am grateful to Dr. Vivek and his team for their professionalism and hospitality.”
This advanced treatment reinforces Dubai’s position as a growing global destination for advanced therapies in the field of rare diseases.
Distributed by APO Group on behalf of Aster DM Healthcare.
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About Medcare Royal Speciality Hospital:
Medcare Royal Speciality Hospital, Al Qusais, part of Aster DM Healthcare, is a 126-bed multi-speciality flagship hospital in Dubai and a recognised regional centre of excellence for rare genetic and neurological disorders. The hospital has treated more than 190 SMA patients with gene therapy, welcoming patients from Egypt, Iran, Turkey, Nepal, Romania, Russia, India, Ethiopia and beyond.
About Itvisma:
Itvisma®️ (onasemnogene abeparvovec-brve), developed by Novartis, is a one-time, fixed-dose intrathecal gene replacement therapy that delivers a functional copy of the SMN1 gene directly into the cerebrospinal fluid. It is approved for SMA patients aged 2 years and older with a confirmed SMN1 mutation.
About SMA:
Spinal Muscular Atrophy is a rare, progressive genetic neuromuscular disorder caused by loss of motor neurons, leading to muscle weakness that can affect movement, breathing and swallowing. Until the approval of Itvisma, gene therapy for SMA was largely restricted to young children.
