Qatar’s Sidra Medicine establishes Gene Therapy Center for rare genetic diseases

Qatar's Sidra Medicine is setting a new benchmark in gene therapy with the launch of its pioneering Gene Therapy Center. The state-of-the-art facility is dedicated to treating rare genetic disorders like Spinal Muscular Atrophy (SMA) and Duchenne Muscular Dystrophy (DMD). The move reinforces Qatar's commitment to cutting-edge medical innovation while offering families in the Middle East and North Africa (MENA) region world-class treatment close to home.
"In response to the overwhelming demand from neighboring countries, the establishment of the Gene Therapy Center is a crucial step in meeting the needs of families seeking cutting-edge treatments for SMA and DMD," said Prof Tawfeg Ben-Omran, division chief of Genetic and Genomic Medicine at Sidra Medicine.
With a focus on advanced medical solutions, Sidra Medicine has already administered life-changing treatments to dozens of children, with ambitious plans to expand its services further.
How does it work?
Gene therapy is rewriting the script for previously untreatable conditions. By targeting the genetic root of diseases like SMA and DMD, these therapies work by introducing functional copies of defective genes directly into patients' cells.
For SMA, a rare but severe neuromuscular disorder, Sidra Medicine administers Zolgensma, a groundbreaking gene therapy developed by Novartis. This one-time infusion halts disease progression and improves motor function, giving children a fighting chance at a normal life. Similarly, for DMD, a debilitating muscle-wasting disorder, Elevidys-a gene therapy developed by Roche-introduces a shortened but functional version of the dystrophin gene, effectively slowing the disease's progression and improving muscle strength.
"The availability of gene therapies for both SMA and DMD at Sidra Medicine represents a significant leap forward in the care of these rare genetic disorders," said Prof Ibrahim Janahi, acting chief medical officer at Sidra Medicine.
Why does it matter?
The implications of these advancements extend far beyond Qatar's borders. Rare diseases often come with not just physical burdens but also immense emotional and financial strain on families. Gene therapy presents a life-altering alternative to long-term palliative care, allowing children to regain motor function and, in many cases, lead significantly improved lives.
Beyond the science, the center is reducing the logistical and emotional toll on families who previously had to travel long distances for treatment.
"By offering these life-changing treatments, we are not only providing advanced medical solutions but also reducing the emotional and logistical challenges families face," added Janahi.
The context
DMD and SMA are among the most devastating inherited conditions, with profound impacts on affected children and their families. SMA, which affects approximately 1 in 10,000 live births, leads to progressive muscle weakness and, if left untreated, can be fatal. Meanwhile, DMD primarily affects boys, with one in every 3,500 to 5,000 male births impacted globally. In Saudi Arabia and other GCC nations, late diagnoses have been a major barrier to timely intervention.
Historically, treatment options were limited to symptom management. But with recent breakthroughs in gene therapy, patients now have a chance at a vastly improved quality of life. Sidra Medicine has positioned itself as a regional leader in these interventions, offering a beacon of hope to families across the MENA region. In 2024, the hospital became the first in Qatar and only the fifth in the world to administer Elevidys for DMD. Later that year, it became the first institution outside the United States to provide this treatment to children over six years old, further cementing its reputation as a leader in the field.
Sidra's International Patient Services has been instrumental in connecting families to these advanced treatments, welcoming over 1,000 international patients from across the globe, including the GCC, Africa, and even South America.
"The diversity of our international patients at Sidra Medicine is a testament to our expertise," said Prof Ziyad Hijazi, medical director of International Patient Services.
With the establishment of its Gene Therapy Center, Sidra Medicine aims to rewrite the future for children with genetic disorders — bringing world-class care to those who need it most.
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