Doha, Qatar: Sidra Medicine, a member of Qatar Foundation, successfully administered a life-saving medication for Malkha Rouhi, a baby diagnosed with Spinal Muscular Atrophy (SMA). The treatment, which offers hope for children with the severe and rare genetic disease, was made possible through the generous fundraising efforts of the broader community in Qatar.
Dr. Tawfeg Ben-Omran, Division Chief of Genetic and Genomic Medicine at Sidra Medicine stated: "We are honored by the trust placed in us by Malkha’s family. Her groundbreaking gene therapy is an example of the power of collaboration between healthcare institutions, Qatar Charity and the community. We are proud that our life-saving treatments can provide a new chance for children facing such devastating diagnoses. We wish Malkha the very best of health and will be supporting her family in her post-recovery care."
Sidra Medicine is the only hospital in Qatar equipped to provide groundbreaking gene therapy for SMA, an inherited neuromuscular disorder that affects nerves and muscles, causing muscles weakness, breathing and swallowing difficulties. The treatment, which has the potential to significantly improve the quality of life for SMA patients, underscores Sidra Medicine's commitment to delivering world-class healthcare and offering cutting-edge genetic therapies for rare diseases.
Dr. Khalid Ibrahim, Senior Attending Pediatric Neurologist at Sidra Medicine said: "It is a privilege to be part of the team that administered Malkha’s infusion. This treatment highlights the incredible advancements in precision medicine, and we are grateful for the opportunity to bring such life-changing care to our young patients. We have successfully administered the medication to nearly 50 babies with SMA at our hospital. These patients have come to us from across the Middle East and have been part of a comprehensive SMA management program that Sidra Medicine established in 2022."
Malkha’s parents said Risal Abdul Rasheed and Nihala Nizam said: "Words cannot express the generosity and kindness of the people in Qatar, Qatar Charity and the wonderful medical team at Sidra Medicine. Alhamdulilah, that we were able to have this life-saving treatment for our baby. Thanks to the support of Qatar Charity, we were able to raise the funds in such a short amount of time. We want to acknowledge the community, for supporting us financially and emotionally, in this journey. We feel incredibly blessed and fortunate to have access to such amazing care in Qatar. Thank you for giving Malkha a chance. We look forward to sharing this beautiful story with Malkha and how everyone came together to save her life. You have our deepest love and gratitude."
Malkha was administered Zolgensma, a lifesaving drug made by Novartis for treating SMA. Sidra Medicine has been appointed as the lead treatment center in the Middle East and North Africa (MENA) region to administer the medication by Novartis.
The successful treatment of Malkha Rouhi reinforces Sidra Medicine's position as a leader in pediatric care for genetic diseases. By collaborating with international institutions and investing in cutting-edge technologies, Sidra Medicine is dedicated to providing the highest quality of care to patients in Qatar.
