Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial.
- DOI
- 10.1089/nat.2021.0043
- Published
- 2022 Feb
- Container
- Nucleic acid therapeutics
- Publisher
- Not recorded
- Open access
- yes
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limited evidence Score 45/100 under policy 1.0.0. This is a metadata assessment, not a judgment of the paper's conclusions.
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Cite this work
BibTeX
@article{allodium:10.1089/nat.2021.0043,
title = {Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial.},
author = {Servais L and Mercuri E and Straub V and Guglieri M and Seferian AM and Scoto M and Leone D and Koenig E and Khan N and Dugar A and Wang X and Han B and Wang D and Muntoni F and SKIP-NMD Study Group},
year = {2022},
journal = {Nucleic acid therapeutics},
doi = {10.1089/nat.2021.0043},
url = {https://doi.org/10.1089/nat.2021.0043}
}RIS
TY - JOUR TI - Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial. AU - Servais L AU - Mercuri E AU - Straub V AU - Guglieri M AU - Seferian AM AU - Scoto M AU - Leone D AU - Koenig E AU - Khan N AU - Dugar A AU - Wang X AU - Han B AU - Wang D AU - Muntoni F AU - SKIP-NMD Study Group PY - 2022 JO - Nucleic acid therapeutics DO - 10.1089/nat.2021.0043 UR - https://doi.org/10.1089/nat.2021.0043 ER -
APA
L, S., E, M., V, S., M, G., AM, S., M, S., D, L., E, K., N, K., A, D., X, W., B, H., D, W., F, M., & Group, S. S. (2022). Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial.. Nucleic acid therapeutics. https://doi.org/10.1089/nat.2021.0043
Source records
- pubmed · retrieved 2026-09-25T18:27:54.109Z