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. 2024 Oct 21:47:101112.
doi: 10.1016/j.lanepe.2024.101112. eCollection 2024 Dec.

Gene therapy for spinal muscular atrophy: timing is key

Laurent Servais  1   2
Affiliations

Gene therapy for spinal muscular atrophy: timing is key

Laurent Servais. Lancet Reg Health Eur. .
No abstract available

PubMed Disclaimer

Conflict of interest statement

LS has given consultancy/lectures for Biogen, Novartis, Roche, BioHaven, Scholar Rock, Zentech and Sysnav. He is part of the Data Safety Monitoring Board of NMB biopharma.

References

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    1. Govoni A., Gagliardi D., Comi G.P., Corti S. Time is motor neuron: therapeutic window and its correlation with pathogenetic mechanisms in spinal muscular atrophy. Mol Neurobiol. 2018;55:6307–6318. - PubMed
    1. Weiß C., Becker L., Friese J., et al. Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH region: a population-based observational study. Lancet Reg Health Eur. 2024;47 doi: 10.1016/j.lanepe.2024.101092. - DOI - PMC - PubMed
    1. Gowda V., Atherton M., Murugan A., et al. Efficacy and safety of onasemnogene abeparvovec in children with spinal muscular atrophy type 1: real-world evidence from six infusion centres in the United Kingdom. Lancet Reg Health Eur. 2024;37 - PMC - PubMed
    1. Vrščaj E, Dangouloff T, Osredkar D, Servais L. Newborn screening programs for spinal muscular atrophy worldwide in 2023. J Neuromuscular Dis. In press. DOI 10.1177/22143602241288095. - PubMed