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Pulmonary Cell NewsResearchers described Qatari experience with onasemnogene abeparvovec by reviewing the clinical outcomes of nine spinal muscular atrophy children.
[Gene Therapy]
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Ali, H. G., Ibrahim, K., Elsaid, M. F., Mohamed, R. B., Abeidah, M. I. A., Al Rawwas, A. O., Elshafey, K., Almulla, H., El-Akouri, K., Almulla, M., Othman, A., Musa, S., Al-Mesaifri, F., Ali, R., Shahbeck, N., Al-Mureikhi, M., Alsulaiman, R., Alkaabi, S., & Ben-Omran, T. (2021). Gene therapy for spinal muscular atrophy: the Qatari experience. Gene Therapy, 1–5. https://doi.org/10.1038/s41434-021-00273-7 Cite
