Doha, July 28 (QNA) - Sidra Medicine has become the first hospital in Qatar to administer Itvisma™ (onasemnogene abeparvovec) an advanced one-time gene replacement therapy to 13-year-old Khalid, an adolescent living with spinal muscular atrophy (SMA). 
The milestone represents an important advancement in Qatar's precision medicine journey, marking it as the third country in the world to administer Itvisma™. The new gene-therapy protocol also highlights Sidra Medicine's leadership in delivering highly specialized therapies for patients living with rare genetic diseases. 
Chief Medical Officer at Sidra Medicine Prof. Ibrahim Janahi said: "Administering Itvisma™ to our first patient marks an important milestone not only for Sidra Medicine, but also for the future of rare disease care in Qatar. It demonstrates what can be achieved when multidisciplinary expertise, precision medicine, and advanced interventional radiology capabilities come together around one goal: improving outcomes for patients with complex genetic conditions. Our priority is to ensure that eligible patients and their families can access advanced therapies closer to home through a coordinated care pathway that supports them before, during, and long after treatment."
Spinal muscular atrophy is a rare genetic neuromuscular disease caused by a missing or non-functioning survival motor neuron 1 (SMN1) gene. Without a functioning SMN1 gene, the body cannot produce enough survival motor neuron (SMN) protein, which is essential for motor neuron survival and normal muscle function.
Itvisma™ is a one-time intrathecal gene replacement therapy designed to address the underlying genetic cause of SMA by delivering a functional copy of the human SMN1 gene. Treatment eligibility is determined following comprehensive clinical and genetic assessment by an experienced multidisciplinary team.
Itvisma™ is registered with the Ministry of Public Health, enabling eligible patients to access the therapy locally following comprehensive specialist assessment. (QNA)