Therapy development for spinal muscular atrophy: perspectives for muscular dystrophies and neurodegenerative disorders

Therapy development for spinal muscular atrophy: perspectives for muscular dystrophies and neurodegenerative disorders

Abstract Background Major efforts have been made in the last decade to develop and improve therapies for proximal spinal muscular atrophy (SMA). The introduction of Nusinersen/Spinraza™ as an antisense oligonucleotide therapy, Onasemnogene abeparvovec/Zolgensma™ as an AAV9-based gene therapy and Ris...

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Bibliographic Details
Main Authors: Sibylle Jablonka, Luisa Hennlein, Michael Sendtner
Format: Article
Language:English
Published: BMC 2022-01-01
Series:Neurological Research and Practice
Subjects:
Motoneuron disease
Neurodegenerative disease
Muscular disease
Spinal muscular atrophy
Amyotrophic lateral sclerosis
Muscular dystrophy
Online Access:https://doi.org/10.1186/s42466-021-00162-9

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