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Combination Therapy With Nusinersen and Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy Type I

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source: Journal of clinical medicine

year: 2021

authors: Mirea A,Shelby ES,Axente M,Badina M,Padure L,Leanca M,Dima V,Sporea C


Spinal muscular atrophy (SMA) is a neuromuscular progressive disease, characterized by decreased amounts of survival motor neuron (SMN) protein, due to an autosomal recessive genetic defect. Despite recent research, there is still no cure. Nusinersen, an antisense oligonucleotide acting on the gene, is intrathecally administered all life long, while onasemnogene abeparvovec-xioi, a gene therapy, is administered intravenously only once. Both therapies have proven efficacy, with best outcomes obtained when administered presymptomatically. In recent years, disease-modifying therapies such as nusinersen and onasemnogene abeparvovec-xioi have changed the natural history of SMA.

organization: Faculty of Midwifery and Nursing, University of Medicine and Pharmacy "Carol Davila", 37 Dionisie Lupu Street, 020021 Bucharest, Romania.

DOI: 10.3390/jcm10235540

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