Tribune News Network
Doha
Sidra Medicine, a member of Qatar Foundation, has established a Gene Therapy Center to treat rare genetic diseases such as Spinal Muscular Atrophy (SMA) and Duchenne Muscular Dystrophy (DMD).
The announcement was made at Arab Health 2025 where Sidra Medicine will highlight its international patient service programmes, including flagship services such as gene therapy, reproductive medicine, the Heart Center and more.
The Gene Therapy Center solidifies Sidra Medicine’s position as a regional leader in advanced care, attracting families from across the GCC and the Middle East and North Africa (MENA) region.
Prof Tawfeg Ben-Omran, division chief of Genetic and Genomic Medicine at Sidra Medicine, said: "In response to the overwhelming demand from neighbouring 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. As a Qatari institution, our goal is to create a regional centre of excellence, where children from across the Arab world can access advanced therapies without the need to travel long distances, bringing world-class care closer to home.”
The Gene Therapy Center is designed to address the growing demand for innovative treatments for SMA and DMD. It plans to expand the service in the future for other rare and complex genetic diseases. Since the launch of its gene therapy programme in 2022, Sidra Medicine has already provided groundbreaking therapies to 30 children with SMA and 15 children with DMD, attracting patients from 11 countries.
Prof Ibrahim Janahi, acting chief medical officer, chair of Medical Education, and division chief of Pulmonology, said: "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. By offering these life-changing treatments, we are not only providing advanced medical solutions but also reducing the emotional and logistical challenges families face. Our International Patient Services team is dedicated to guiding families through every step of their journey, ensuring a smooth and efficient experience throughout their time with us.”
Leading the Way in Duchenne Muscular Dystrophy (DMD) Care
DMD is the most common inherited muscle disorder, primarily affecting males, with symptoms typically appearing between the ages of two and three. As the disease progresses, it leads to severe muscle weakness, affecting mobility and, ultimately, respiratory and cardiac function. Globally, approximately one in 3,500 to 5,000 live male births are affected by the disorder. In the GCC region, specifically Saudi Arabia, studies have indicated a higher average age at diagnosis (7-8 years) compared to countries such as the United Kingdom – where the mean age is slightly above four years. This delay is attributed to factors such as limited awareness and lack of standardised diagnostic pathways.
Recent advancements in gene therapy have provided new hope for DMD patients. Elevidys, developed by Roche Pharmaceuticals, is the first and only approved gene therapy for DMD. The treatment introduces a functional dystrophin gene to muscle and heart cells, slowing disease progression and improving muscle function.
Sidra Medicine has become a key player in DMD care in the region. In June 2024, it made history by becoming the first hospital in Qatar and the fifth hospital worldwide to administer Elevidys to a DMD patient, Chris El Kik. This milestone marked the beginning of Sidra Medicine’s rapidly expanding DMD programme.
Building on this success, Sidra Medicine achieved another groundbreaking milestone in October 2024, becoming the first hospital outside the United States to administer Elevidys to children over the age of six. This highlights Sidra Medicine’s growing expertise and leadership in the care and treatment of DMD.
Advancing Treatment for Spinal Muscular Atrophy (SMA)
SMA is a rare genetic disorder that causes progressive muscle weakness and can be life-threatening, often resulting in the loss of vital motor functions such as sitting, crawling, or swallowing. In Saudi Arabia, the SMA carrier frequency is reported at 2.6 percent, which is slightly higher than the global average of 1.25 percent to two percent.
The estimated birth incidence of SMA in Saudi Arabia is 32 per 100,000 live births, with approximately 2,265 individuals currently living with the condition. However, with advances in gene therapy, SMA is now treatable with Zolgensma, a cutting-edge treatment developed by Novartis that halts disease progression and improves outcomes.
Sidra Medicine was selected as the lead treatment centre for the MENA region by Novartis and has successfully administered Zolgensma to several children, including Malkha Rouhi, a young girl who received the therapy at the hospital.
International Patient Services
Each year, Sidra Medicine’s International Patient Services team works with families from outside Qatar who travel to receive specialised care at the hospital.
Prof Ziyad Hijazi, medical director of International Patient Services and senior attending cardiologist, said: "The diversity of our international patients at Sidra Medicine is a testament to our expertise.
To date, the hospital has cared for over 1,000 international patients from around the world, including Kuwait, Afghanistan, Iran, Iraq, Oman, Egypt, Turkey, Mozambique, Uganda, Kenya, South America, the United Kingdom and more. We continue to be a trusted destination for those seeking the latest in paediatric cardiovascular care, neurosurgery, neurology, urology and gene therapy treatments including SMA and DMD.”
If you are based overseas and want to inquire about our services including the Gene Therapy Center, please contact International.services@sidra.org. If you are based in Qatar, please contact 40033333 for more information.