Medcare Royal Specialty Hospital becomes first outside US to offer adult SMA gene therapy in Dubai

UAE—Medcare Royal Specialty Hospital (MRSH) in Dubai has become the first hospital outside the United States to provide Itvisma, a newly licensed intrathecal gene therapy, to adult patients living with Spinal Muscular Atrophy (SMA).

The milestone expands access to advanced treatment for older patients who previously had limited therapeutic options.

The hospital recently administered the one-time treatment to a 22-year-old Egyptian patient who was diagnosed with SMA at 18 months of age and has relied on a wheelchair for much of his life.

Last year, Medcare also became the first healthcare provider worldwide to administer the newly approved therapy to a four-year-old international SMA patient, strengthening its position in the field of rare disease treatment.

Expanding access for older SMA patients

SMA is a rare inherited neuromuscular disorder that causes progressive muscle weakness and reduced mobility.

As the disease advances, it can also affect breathing, swallowing, and other essential body functions.

Until recently, major advances in SMA gene therapy were largely limited to children under the age of two.

However, Medcare is now offering treatment to patients older than two years through the newly approved therapy, giving hope to adolescents and adults who were previously excluded from such innovations.

Dr. Shanila Laiju, Group CEO of Medcare Hospitals and Medical Centres, said expanding SMA treatment to adults could change the lives of thousands of patients across the Middle East and beyond.

She added that the introduction of Itvisma builds on Medcare’s earlier work in treating older SMA patients, a contribution that has been recognized by Novartis.

Regional referral centre for rare disease care

Medcare continues to attract patients from across multiple regions seeking advanced therapies.

According to the hospital, it has treated more than 190 SMA patients and 20 Duchenne Muscular Dystrophy (DMD) patients from the Middle East, Africa, Asia, and Europe.

Globally, SMA affects about one in every 10,000 live births, while DMD impacts an estimated 300,000 boys worldwide.

Multidisciplinary team delivered therapy

A specialist multidisciplinary team carried out the treatment at Medcare.

The team was led by Gene Therapy Unit Clinical Lead Dr. Vivek Mundada and included adult neurologist Dr. Sagar Kawale, anaesthesia specialists Dr. Neha Shahane, and Dr. Ardalan Papari.

Dr. Mundada said adult SMA patients had long lacked access to curative treatment options.

He explained that Itvisma works by delivering a functional copy of the missing SMN1 gene directly into the cerebrospinal fluid, targeting the underlying cause of the condition.

He added that the therapy opens new possibilities for many patients who previously had little hope, noting that the recent patient travelled from Egypt to Dubai specifically to receive the treatment.

Patient shares experience

Following the procedure, the patient described the treatment experience as comfortable and positive.

He said he felt no pain around the injection site and was relieved that he would no longer need the multiple injections he had previously undergone.