Treatment of spinal muscular atrophy with Onasemnogene Abeparvovec in Switzerland: a prospective observational case series study

Treatment of spinal muscular atrophy with Onasemnogene Abeparvovec in Switzerland: a prospective observational case series study

Abstract Background Spinal muscular atrophy (SMA) is a rare neuromuscular disorder leading to early death in the majority of affected individuals without treatment. Recently, targeted treatment approaches including Onasemnogene Abeparvovec (OA) were introduced. This study describes the first real-wo...

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Bibliographic Details
Main Authors: Georg M. Stettner, Oswald Hasselmann, Anne Tscherter, Elea Galiart, David Jacquier, Andrea Klein
Format: Article
Language:English
Published: BMC 2023-02-01
Series:BMC Neurology
Subjects:
Spinal muscular atrophy
Onasemnogene Abeparvovec
Zolgensma
Gene therapy
Switzerland
Online Access:https://doi.org/10.1186/s12883-023-03133-6

Internet

https://doi.org/10.1186/s12883-023-03133-6

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