| Autor: |
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 |
| Rok vydání: |
2022 |
| Předmět: |
|
| DOI: |
10.57945/manara.21598068 |
| Popis: |
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. Children License: https://creativecommons.org/licenses/by/4.0See article on publisher's website: http://dx.doi.org/10.1038/s41434-021-00273-7 |
| Databáze: |
OpenAIRE |
| Externí odkaz: |
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