Gene therapy for spinal muscular atrophy: the Qatari experience

Hossamaldein Gaber Ali, Khalid Ibrahim, Mahmoud Fawzi Elsaid, Reem Babiker Mohamed, Mahmoud I. A. Abeidah, Azhar Othman Al Rawwas, Khaled Elshafey, Hajer Almulla, Karen El-Akouri, Mariam Almulla, Amna Othman, Sara Musa, Fatma Al-Mesaifri, Rehab Ali, Noora Shahbeck, Mariam Al-Mureikhi, Reem Alsulaiman, Saad Alkaabi & Tawfeg Ben-Omran
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder characterized by hypotonia, progressive muscle weakness, and wasting. Onasemnogene abeparvovec (Zolgensma®) is a novel gene therapy medicine, FDA-approved in May 2019 for the treatment of SMA. This study aimed to describe Qatari experience with onasemnogene abeparvovec by reviewing the clinical outcomes of 9 SMA children (7 SMA type 1 and 2 with SMA type 2) aged 4‒23 months treated between November 2019 and July 2020....
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