Dubai hospital pioneers adult SMA gene therapy breakthrough outside US

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Adult SMA gene therapy breakthrough in Dubai marks global milestone, offering life-changing treatment and expanding access beyond children for the first time.

DUBAI: Adult SMA gene therapy breakthrough has been achieved in Dubai, where Medcare Royal Specialty Hospital treated a 22-year-old patient with a one-time gene therapy, marking the first such case outside the United States. The milestone signals a major shift in how spinal muscular atrophy is treated globally.

The patient, who travelled from Egypt, received the newly licensed therapy Itvisma, developed to address the root genetic cause of the condition. Spinal muscular atrophy is a rare disorder caused by a defective SMN1 gene, leading to progressive muscle weakness and loss of mobility. Until now, advanced gene therapies were largely limited to infants and young children, leaving adult patients with limited options.

This adult SMA gene therapy breakthrough expands access to life-changing treatment for a wider patient group. Medical experts highlighted that the procedure required a highly specialised, multidisciplinary approach due to complications such as prior spinal surgery, making the successful delivery particularly significant.

Medcare officials said the achievement reflects the UAE’s growing leadership in advanced healthcare and innovation. The hospital has already treated more than 190 SMA patients and continues to attract patients from across the Middle East, Europe, Asia and Africa.

Doctors involved in the case described the development as a turning point in neuromuscular care, opening new possibilities for patients previously excluded from cutting-edge therapies. The one-time treatment replaces the need for repeated injections, offering long-term clinical benefits and improved quality of life.

The breakthrough also reinforces Dubai’s position as a global destination for specialised medical care, particularly in rare diseases and gene therapy. Experts say such advancements will play a key role in shaping the future of personalised medicine and expanding equitable access to innovative treatments worldwide.

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