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Article

Gene therapy for spinal muscular atrophy: the Qatari experience.

Gene therapy - 1 Nov 2021

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

Abstract excerpt

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...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 34276047
DOI: 10.1038/s41434-021-00273-7
PMCID: PMC8599021

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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