Gene therapy for spinal muscular atrophy: the Qatari experience

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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Bibliographic Details
Main Author: Hossamaldein Gaber Ali (14152632) (author)
Other Authors: 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)
Published: 2021
Subjects:
Biological sciences
Genetics
Biomedical and clinical sciences
Clinical sciences
Neurosciences
Chemical sciences
Medicinal and biomolecular chemistry
Gene therapy
Neurological disorders
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