Sidrā introduces first spinal muscular atrophy gene therapy
Sidra Medicine in Qatar has successfully administered the first gene therapy for spinal muscular atrophy, making it one of the first hospitals worldwide to offer this treatment. The therapy, given as a one-time injection into the cerebrospinal fluid, aims to correct the genetic cause of the disease by delivering a healthy copy of the SMN1 gene. Eligible patients can receive this treatment after a comprehensive evaluation by a multidisciplinary medical team. This achievement represents a significant step forward in the development of medical care for rare genetic diseases in Qatar and enhances the country's reputation as a leading center for delivering specialized treatments.
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