Trusted Resources: Education
Scientific literature and patient education texts
Combination Therapy With Nusinersen and Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy Type I
source: Journal of clinical medicine
year: 2021
authors: Mirea A,Shelby ES,Axente M,Badina M,Padure L,Leanca M,Dima V,Sporea C
summary/abstract: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
read more
Related Content
-
Spinal Muscular Atrophy Fact SheetWhat is spinal muscular atrophy?Spinal m...
-
Biogen Announces New Updates Across Its SMA Research Program at 2022 MDA ConferenceBiogen Inc. announced new data and updat...
-
Intrathecal Administration of Nusinersen Using the Ommaya Reservoir in an Adult With 5q-Related Spinal Muscular Atro...Spinal muscular atrophy (SMA) is a hered...
-
SMA and Nusinersen (Spinraza) the Story so farhttps://slideplayer.com/slide/14664651/...
-
Stanford Healthcare – Spinal Muscular AtrophyStanford Neuromuscular Program has exten...
-
Why Some Drugs Cost $2.1 Million per Dose and how one Company Plans to Change ThisWith a cost of $2.1 million for a one-do...
-
Despite Taking Spinraza, SMA Type 1 Children Show Nonmotor ProblemsDespite its well-reported benefits in mo...