Dubai, UAE – April 28, 2026: 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.