UAE milestone broadens access to life-changing treatment for adults.
Dubai: Dubai has marked a major global medical breakthrough by successfully administering a newly licensed gene therapy to an adult patient with Spinal Muscular Atrophy (SMA), becoming the first location outside the United States to do so.
The treatment was delivered to a 22-year-old Egyptian patient at Medcare Royal Specialty Hospital. The patient had spent most of his life in a wheelchair with limited therapeutic options.
His case represents only the second time worldwide that an adult SMA patient has received this advanced one-time gene therapy, according to Novartis, the manufacturer.
The milestone marks a turning point in the treatment of Spinal Muscular Atrophy, a rare genetic neuromuscular disorder that causes progressive muscle weakness and can severely affect mobility, breathing, and swallowing.
Historically, gene therapy for SMA has been largely limited to infants and children under the age of two, leaving adult patients with very few effective treatment options.
Older patients
This latest development changes that trajectory, opening the door to broader access to advanced therapies for older patients.
Spinal Muscular Atrophy is caused by a faulty or missing SMN1 gene, which is essential for producing a protein that supports motor neuron survival. Without it, patients experience gradual and often severe muscle degeneration.
Highly complex
Dr Shanila Laiju, Group CEO of Medcare Hospitals, said expanding treatment capabilities to adults could transform outcomes for thousands of patients.
“We believe that achieving the capabilities to provide Spinal Muscular Atrophy treatment to adults will rewrite the life stories of thousands of patients in the region and beyond. Expanding access to advanced therapies for older patients is a meaningful step toward equity in care,” she said.
The procedure itself was highly complex, requiring a coordinated, multidisciplinary approach because of the patient’s previous spinal fusion surgery for scoliosis — a common complication in SMA cases that changes spinal anatomy and makes access to the spinal canal especially difficult.
Despite these challenges, the medical team successfully administered the one-time therapy, known as Itvisma, which works by delivering a functional copy of the SMN1 gene directly into the cerebrospinal fluid, targeting the root cause of the disease.
Dr Vivek Mundada, Clinical Lead of the Gene Therapy Unit, described the achievement as a major step forward in expanding access to advanced care.
“For years, adults with Spinal Muscular Atrophy 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 opens new possibilities for many patients who were previously left without hope,” he said.
He added that the UAE’s progressive healthcare ecosystem has played a key role in enabling such advancements, helping position Dubai as an emerging global hub for rare disease treatment and gene therapy.
Meaningful treatment
Dr Sagar Kawale, an adult neurologist involved in the case, said the development reflects a broader shift in how Spinal Muscular Atrophy is treated worldwide.
“This is a shift in how we approach Spinal Muscular Atrophy care. We are now able to offer meaningful treatment options to adult patients who were previously excluded. It opens the door for many more patients to benefit from gene therapy,” Dr Sagar Kawale said.
The patient, who was diagnosed at just 18 months old, had previously relied on regular intrathecal injections as part of a lifelong treatment plan. Moving to a one-time therapy marks a major improvement in both quality of life and long-term care.
“The experience was very positive. The team was extremely supportive, and the procedure itself was smooth and painless. I am relieved that I no longer need to undergo repeated injections,” the patient said.
Gene therapy
The latest case builds on earlier milestones in gene therapy, including the successful treatment of younger patients and the expansion of eligibility criteria beyond traditional limits.
Globally, Spinal Muscular Atrophy affects approximately 1 to 2 in 100,000 people, while Duchenne muscular dystrophy, another genetic condition treated through similar therapies, affects around one in 3,500 boys.
Since 2020, more than 190 SMA patients and 20 DMD patients have received advanced gene therapies in Dubai, attracting patients from across the Middle East, Europe, Asia, and Africa.
The breakthrough further strengthens Dubai’s growing reputation as a leading destination for advanced medical innovation and specialised care, particularly in the treatment of rare genetic diseases.
