The newly licensed intrathecal gene therapy, enhances life-changing treatment options for children and families affected by Spinal Muscular Atrophy

Dubai, UAE–22December 2025: Families who were previously informed that their children had outgrown the eligibility for SMA gene therapy or were deemed not the right weight, now have renewed hope. Medcare Royal Speciality Hospital in Al Qusais has become among the first centres outside the USA to offer a newly licensed intrathecal gene therapy for Spinal Muscular Atrophy (SMA).

This treatment can now be administered to SMA patients well into their childhood and beyond. Approved after a thorough global assessment for intrathecal use, this therapy is tailored for children, teenagers, and adults, effectively removing the weight restrictions that have historically hindered access to gene therapy for this rare neuromuscular disorder.The milestone solidifies the UAE’s position at the forefront of cutting-edge genetic medicine and brings new hope to thousands of families worldwide.

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