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

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

Background: 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...

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Bibliographic Details
Main Authors: Andrada Mirea, Elena-Silvia Shelby, Mihaela Axente, Mihaela Badina, Liliana Padure, Madalina Leanca, Vlad Dima, Corina Sporea
Format: Article
Language:English
Published: MDPI AG 2021-11-01
Series:Journal of Clinical Medicine
Subjects:
spinal muscular atrophy
combined modifying therapy
nusinersen
onasemnogene abeparvovec-xioi
early treatment
motor evolution
Online Access:https://www.mdpi.com/2077-0383/10/23/5540

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https://www.mdpi.com/2077-0383/10/23/5540

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