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Gene therapy for spinal muscular atrophy: the Qatari experience

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 descri...

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Detalles Bibliográficos
Autores principales:	Ali, Hossamaldein Gaber, Ibrahim, Khalid, Elsaid, Mahmoud Fawzi, Mohamed, Reem Babiker, Abeidah, Mahmoud I. A., Al Rawwas, Azhar Othman, Elshafey, Khaled, Almulla, Hajer, El-Akouri, Karen, Almulla, Mariam, Othman, Amna, Musa, Sara, Al-Mesaifri, Fatma, Ali, Rehab, Shahbeck, Noora, Al-Mureikhi, Mariam, Alsulaiman, Reem, Alkaabi, Saad, Ben-Omran, Tawfeg
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group UK 2021
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8599021/ https://www.ncbi.nlm.nih.gov/pubmed/34276047 http://dx.doi.org/10.1038/s41434-021-00273-7

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