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pro vyhledávání: '"Mahmoud I A, Abeidah"'
Autor:
Hossamaldein Gaber, Ali, Khalid, Ibrahim, Mahmoud Fawzi, Elsaid, Reem Babiker, Mohamed, Mahmoud I A, Abeidah, Azhar Othman, Al Rawwas, Khaled, Elshafey, Hajer, Almulla, Karen, El-Akouri, Mariam, Almulla, Amna, Othman, Sara, Musa, Fatma, Al-Mesaifri, Rehab, Ali, Noora, Shahbeck, Mariam, Al-Mureikhi, Reem, Alsulaiman, Saad, Alkaabi, Tawfeg, Ben-Omran
Publikováno v:
Gene Therapy
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
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=pmid________::afbc230f4178c261b8fa0ddc3ce884d4
https://pubmed.ncbi.nlm.nih.gov/34276047
https://pubmed.ncbi.nlm.nih.gov/34276047
