Sidra is fifth hospital in world to administer gene-therapy for Duchenne-Muscular-Dystrophy

Published: 2 July 2024

NT Bureau
Doha

Sidra Medicine, a member of Qatar Foundation, has become the first hospital in Qatar and the fifth hospital worldwide to administer Elevidys, a revolutionary gene therapy medication for treating Duchenne Muscular Dystrophy (DMD).

DMD is the most common form of inherited muscle disorders. It primarily affects males, with a prevalence of approximately 1 in 3,500 live male births.

Children with DMD typically appear normal at birth, but between the ages of two and three, begin to exhibit developmental delays, particularly in their motor skills and experience difficulties with walking, talking, jumping, or climbing stairs.

Elevidys is currently the only gene therapy medication available for treating DMD. It works by delivering a functional dystrophin gene into the muscle and heart cells. The process slows disease progression and enhances muscle function.

The drug was secured through Ebn Sina Medical, a local agent for Roche Pharmaceuticals, which is responsible for making Elevidys. It was cleared by the U.S. Food and Drug Administration (FDA) in June 2024, for a wide range of DMD patients, including children aged four and older.

Dr. Tawfeg Ben-Omran, the Division Chief of Genetics and Genomic Medicine at Sidra Medicine said: “We are one of the few hospitals in the region with an extensive gene therapy program, reflecting our commitment to providing innovative, safe and effective treatments for genetic and rare diseases. “
Sidra Medicine administered Elevidys to a young international patient from Lebanon who became the first recipient of the drug in Qatar.

Sidra Medicine is in the process of preparing three patients from Qatar and two young international patients from Kuwait to receive the same drug.

The gene therapy program under the Genetic and Genomic Medicine clinic at Sidra Medicine is dedicated to providing life-saving treatments for rare genetic disorders.