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. 2023 Dec 22:37:100822.
doi: 10.1016/j.lanepe.2023.100822. eCollection 2024 Feb.

Gene replacement therapy in spinal muscular atrophy: filling the data gaps

Tim Hagenacker  1 , Ulrike Schara-Schmidt  2
Affiliations

Gene replacement therapy in spinal muscular atrophy: filling the data gaps

Tim Hagenacker et al. Lancet Reg Health Eur. .
No abstract available

PubMed Disclaimer

Conflict of interest statement

Tim Hagenacker declares institutional research support from Novartis, not related to the study. Ulrike Schara-Schmidt declares speaker fees, travel support and advisory board compensation from Novartis.

References

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    1. Mercuri E., Muntoni F., Baranello G., et al. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial. Lancet Neurol. 2021;20(10):832–841. - PubMed
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