Doha: Sidra Medicine has announced a medical achievement by facilitating access to sepiapterin for patients with Phenylketonuria (PKU), making Qatar the third country in the world — after Germany and the United States — to provide this treatment through approved and regulated clinical and governance pathways. This milestone follows the successful administration of sepiapterin to a 12-year-old Qatari girl diagnosed with PKU, a rare inherited metabolic disorder that prevents the body from breaking down the amino acid phenylalanine.
PKU affects an estimated 1 in 23,930 newborns worldwide, with higher incidence reported in parts of Europe and the Middle East.
Prof. Tawfeg Ben-Omran, Division Chief of Genetics and Genomic Medicine at Sidra Medicine, said: “Providing access to sepiapterin for patients with PKU represents a transformative step in metabolic care. This treatment can significantly reduce the lifelong burden of strict dietary therapy and improve quality of life for children and adults living with this condition.”
The achievement marks another important milestone for Sidra Medicine’s Gene Therapy Center, launched in January 2025 under the Genetic and Genomic Medicine Clinic.
