Gene therapy for spinal muscular atrophy: the Qatari experience

<p dir="ltr">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....

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التفاصيل البيبلوغرافية
المؤلف الرئيسي:	Hossamaldein Gaber Ali (14152632) (author)
مؤلفون آخرون:	Khalid Ibrahim (3853360) (author), Mahmoud Fawzi Elsaid (14152635) (author), Reem Babiker Mohamed (14152638) (author), Mahmoud I. A. Abeidah (14152641) (author), Azhar Othman Al Rawwas (14152644) (author), Khaled Elshafey (14152647) (author), Hajer Almulla (14152650) (author), Karen El-Akouri (14152653) (author), Mariam Almulla (14152656) (author), Amna Othman (14152659) (author), Sara Musa (14152662) (author), Fatma Al-Mesaifri (14152665) (author), Rehab Ali (14152668) (author), Noora Shahbeck (14152671) (author), Mariam Al-Mureikhi (7317614) (author), Reem Alsulaiman (14152674) (author), Saad Alkaabi (14152677) (author), Tawfeg Ben-Omran (6663634) (author)
منشور في:	2021
الموضوعات:	Biological sciences Genetics Biomedical and clinical sciences Clinical sciences Neurosciences Chemical sciences Medicinal and biomolecular chemistry Gene therapy Neurological disorders
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Gene therapy for spinal muscular atrophy: the Qatari experience

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