Sidra Medicine pioneers gene therapy in Qatar for children with rare genetic disorders

Doha, Qatar: Sidra Medicine s Genetic and Genomic Medicine clinic is providing life altering gene therapy for rare genetic disorders. In a major achie...

Doha, Qatar: Sidra Medicine’s Genetic and Genomic Medicine clinic is providing life-altering gene therapy for rare genetic disorders. In a major achievement, the hospital became the first outside United States, to administer Elevidys gene therapy to four Qatari children with Duchenne Muscular Dystrophy (DMD), who were over the age of six.

DMD is a severe genetic disorder characterized by progressive muscle degeneration and weakness, primarily affecting boys. The treatment was initially available for children below the age of six years. With the expansion of Sidra Medicine’s DMD programme, the treatment now provides hope for older children with the condition.

“Sidra Medicine has recently successfully administrated of Elevidys to four children aged eight, nine, ten, and eleven years old,” Division Chief of Genetic and Genomic Medicine at Sidra Medicine, Dr. Tawfeg Ben-Omran told The Peninsula.

With an estimated prevalence of 1 in 3,500 live male births, DMD is one of the most severe forms of inherited and common muscular dystrophies. While appearing normal at birth, male children with DMD typically show developmental delays in motor skills between the ages of two and three. This can manifest as difficulties with walking, talking, jumping, or climbing stairs.

“Elevidys, a groundbreaking gene therapy developed by Roche Pharmaceuticals, is currently the only treatment of its kind for DMD. It works by introducing a functional dystrophin gene into muscle and heart cells, aiming to slow disease progression and improve muscle function,” said Dr. Ben-Omran.