Sidra Medicine launches gene therapy center to treat rare diseases

QATAR—Sidra Medicine, a leading healthcare institution in Qatar and a member of Qatar Foundation, has launched a Gene Therapy Center aimed at treating rare genetic diseases, including Spinal Muscular Atrophy (SMA) and Duchenne Muscular Dystrophy (DMD).

This announcement was made during Arab Health 2025, where Sidra showcased its diverse international patient services, including gene therapy, reproductive medicine, and its renowned Heart Center.

The Gene Therapy Center strengthens Sidra Medicine’s position as a regional leader in advanced healthcare, attracting families from across the GCC and MENA regions.

The launch comes just a month after Sidra conducted Qatar’s first industry-sponsored trial for pediatric congenital hyperinsulinism, further demonstrating its commitment to cutting-edge treatments.

Prof. Tawfeg Ben-Omran, the division chief of Genetic and Genomic Medicine at Sidra, emphasized that the center was established in response to the high demand for treatments for SMA and DMD, particularly from neighboring countries.

The center aims to be a regional hub of excellence, providing world-class therapies to children from across the Arab world without the need to travel abroad.

Since launching its gene therapy program in 2022, Sidra Medicine has already provided innovative treatments to 30 children with SMA and 15 children with DMD, drawing patients from 11 different countries.

Prof. Ibrahim Janahi, Sidra’s acting chief medical officer, highlighted the significant impact of gene therapies in treating these complex disorders, which not only offer advanced medical solutions but also ease the emotional and logistical burdens on families.

DMD, the most common inherited muscle disorder affecting mostly boys, causes progressive muscle weakness that can lead to severe mobility and respiratory challenges.

 In the GCC region, studies show that diagnosis often occurs at a later age compared to countries like the UK, primarily due to limited awareness and diagnostic practices.

However, gene therapies like Elevidys, developed by Roche Pharmaceuticals, offer hope for DMD patients by introducing a functional gene that improves muscle function and slows disease progression.

Sidra Medicine became the first hospital in Qatar and the fifth globally to administer Elevidys, and later made history in October 2024 by treating children over six years old, further cementing its expertise.

SMA, another genetic disorder that causes severe muscle weakness, is now treatable with Zolgensma, a therapy developed by Novartis.

Sidra was selected as the lead treatment center for the MENA region and has successfully administered this groundbreaking treatment to several children, offering new hope to families affected by SMA.

Sidra’s International Patient Services team works diligently to assist families traveling from around the world to receive specialized care.

To date, Sidra Medicine has treated over 1,000 international patients from regions including Kuwait, Afghanistan, Iran, Iraq, and South America.

Prof. Ziyad Hijazi, the medical director of International Patient Services, noted that the hospital’s diverse patient base is a testament to its growing reputation and expertise in fields like pediatric cardiovascular care, neurosurgery, and gene therapy.

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