The treatment, which offers hope for children with the severe and rare genetic disease, was made possible through the generous fundraising efforts of Qatar Charity (QC) and the broader community in Qatar.
Dr Tawfeg Ben-Omran, division chief of Genetic and Genomic Medicine at Sidra Medicine stated: "We are honoured 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 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 has the potential to significantly improve the quality of life for SMA patients.
Dr Khalid Ibrahim, senior attending paediatric neurologist at Sidra Medicine said: "This treatment highlights the incredible advancements in precision medicine. 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."
Ahmad Yousef Fakhroo, CEO's assistant for the Resources Development and Communication sector at QC, said: "We are deeply honored to have contributed to providing baby Malkha Rouhi with a life-saving treatment to combat SMA, thanks to the overwhelming support from our community and partners. This successful treatment exemplifies the incredible impact we can achieve when we come together for a common cause."
Malkha’s parents 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 centre in the Mena region to administer the medication by Novartis.
Fakhro concluded: "Qatar Charity remains committed to its mission of providing life-saving support and bringing hope and healing to families in need. We thank everyone who contributed to providing the life-saving treatment to Malkha Rouhi. And we hope for their continued support for humanitarian causes in the future."
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