Doha, Qatar – Roche Pharmaceuticals, in partnership with Ebn Sina Medical (a subsidiary of Aamal Company Q.P.S.C), recently hosted the Duchenne Muscular Dystrophy (DMD) Peer-to-Peer Exchange at Sidra Medicine. The event brought together 25 leading neurologists from nine countries to share expertise and learn from Sidra Medicine’s advancements in rare disease treatment. This initiative underscores Qatar’s growing role as a regional hub for innovative pediatric healthcare and gene therapy.
The two-day exchange, held at Sidra Medicine, focused on practical applications of advanced therapies for Duchenne Muscular Dystrophy. Participants engaged directly with Sidra Medicine’s Gene Therapy Team, discussing site readiness, patient management, and multidisciplinary coordination. The event highlights a collaborative effort to improve outcomes for patients with this rare genetic disorder.
Advancing DMD Treatment Through Global Collaboration
Duchenne Muscular Dystrophy is a progressive genetic disorder primarily affecting males, causing muscle weakness and loss. According to the Centers for Disease Control and Prevention, it affects approximately 1 in 3,500-5,000 male births. Traditional treatment options have focused on managing symptoms, but recent advancements in gene therapy offer potential for more transformative interventions.
Sidra Medicine established a dedicated Gene Therapy Centre earlier in 2025, solidifying its position as a leader in this emerging field. Since 2024, the hospital has provided gene therapy protocols to 15 children from multiple countries, demonstrating its commitment to international collaboration and access to cutting-edge treatments. This initiative is part of a broader effort to establish Qatar as a center of excellence for pediatric gene therapy.
Sidra Medicine’s Role in Rare Disease Care
Prof. Tawfeg Ben-Omran, Division Chief of Genetic and Genomic Medicine at Sidra Medicine, emphasized the importance of knowledge sharing. “The Peer-to-Peer DMD Exchange programme with Roche highlights Sidra Medicine’s growing expertise and leadership in the care and treatment of DMD,” he stated. The exchange aims to address the need for standardized best practices in implementing innovative treatment protocols globally.
Roche’s Gulf Cluster Head, Dr. Ahmed Tawfik, echoed this sentiment, noting the company’s dedication to supporting scientific exchange. “At Roche, we are deeply committed to improving the lives of patients with rare diseases,” he said. “True progress comes from collaboration, and Sidra Medicine is a true centre of excellence.”
Aamal Company’s Support for Healthcare Innovation
The event also received support from Aamal Company Q.P.S.C., whose subsidiary, Ebn Sina Medical, partnered with Roche to facilitate the exchange. Rashid bin Ali Al Mansouri, CEO of Aamal Company, highlighted the initiative’s contribution to Qatar’s healthcare sector. “Such initiatives contribute to the exchange of expertise among leading specialists and strengthen Qatar’s position as a hub for advanced healthcare and medical innovation,” he commented.
Additionally, the focus on gene therapy represents a significant shift in the approach to treating genetic diseases. While still relatively new, gene therapy holds the promise of correcting the underlying genetic defects that cause conditions like DMD, offering the potential for long-term benefits. However, challenges remain in terms of cost, accessibility, and long-term safety monitoring.
The exchange also touched upon the importance of pediatric neurology and the specialized care required for children with neuromuscular disorders. Effective management of DMD requires a multidisciplinary team, including neurologists, geneticists, physical therapists, and other healthcare professionals.
Looking ahead, Sidra Medicine plans to continue expanding its gene therapy services and fostering international collaborations. The long-term impact of these efforts on patient outcomes remains to be seen, but the recent exchange represents a positive step towards advancing the field of Duchenne Muscular Dystrophy treatment and solidifying Qatar’s position as a regional leader in innovative healthcare. Further research and data collection will be crucial to assess the efficacy and safety of these emerging therapies.
Roche Pharmaceuticals
Sidra Medicine
Aamal Company Q.P.S.C.
CDC – Duchenne Muscular Dystrophy
