The DOI matches its Crossref record; 7 of the 7 references it cites were found in a registry or an index.
Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial
Open accessAbstract
SPR1NT (NCT03505099) was a Phase III, multicenter, single-arm study to investigate the efficacy and safety of onasemnogene abeparvovec for presymptomatic children with biallelic SMN1 mutations treated at ≤6 weeks of life. Here, we report final results for 14 children with two copies of SMN2, expected to develop spinal muscular atrophy (SMA) type 1. Efficacy was compared with a matched Pediatric Neuromuscular Clinical Research natural-history cohort (n = 23). All 14 enrolled infants sat independently for ≥30 seconds at any visit ≤18 months (Bayley-III item #26;P < 0.001; 11 within the normal developmental window). All survived without permanent ventilation at 14 months as per protocol; 13 maintained body weight (≥3rd WHO percentile) through 18 months. No child used nutritional or respiratory support. No serious adverse events were considered related to treatment by the investigator. Onasemnogene abeparvovec was effective and well-tolerated for children expected to develop SMA type 1, highlighting the urgency for universal newborn screening.
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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
Mendell, J. R., Al-Zaidy, S., Shell, R., Arnold, W. D., Rodino-Klapac, L. R., Prior, T. W., Lowes, L., Alfano, L., Berry, K., Church, K., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1706198 · Cited by 2,381
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Foust, K. D., Nurre, E., Montgomery, C. L., Hernandez, A., Chan, C. M., & Kaspar, B. K. (2009) · Nature Biotechnology · doi:10.1038/nbt.1515 · Cited by 1,456
Day, J. W., Finkel, R. S., Chiriboga, C. A., Connolly, A. M., Crawford, T. O., Darras, B. T., Iannaccone, S. T., Kuntz, N. L., Peña, L. D. M., Shieh, P. B., et al. (2021) · The Lancet Neurology · doi:10.1016/s1474-4422(21)00001-6 · Cited by 494
Self-citationConsolidated principles for screening based on a systematic review and consensus process
Dobrow, M. J., Hagens, V., Chafe, R., Sullivan, T., & Rabeneck, L. (2018) · Canadian Medical Association Journal · doi:10.1503/cmaj.171154 · Cited by 359
Chand, D., Mohr, F., McMillan, H., Tukov, F. F., Montgomery, K., Kleyn, A., Sun, R., Tauscher-Wisniewski, S., Kaufmann, P., & Kullak-Ublick, G. (2021) · Journal of Hepatology · doi:10.1016/j.jhep.2020.11.001 · Cited by 306
Self-citationMendell, J. R., Al-Zaidy, S. A., Lehman, K. J., McColly, M., Lowes, L. P., Alfano, L. N., Reash, N. F., Iammarino, M. A., Church, K. R., Kleyn, A., et al. (2021) · JAMA Neurology · doi:10.1001/jamaneurol.2021.1272 · Cited by 295
Self-citationAl-Zaidy, S. A., Kolb, S. J., Lowes, L., Alfano, L. N., Shell, R., Church, K. R., Nagendran, S., Sproule, D. M., Feltner, D. E., Wells, C., et al. (2019) · Journal of Neuromuscular Diseases · doi:10.3233/jnd-190403 · Cited by 185
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Creatine monohydrate supplementation for older adults and clinical populations
Candow, D. G., Ostojic, S. M., Chilibeck, P. D., Longobardi, I., Gualano, B., Tarnopolsky, M. A., Wallimann, T., Moriarty, T., Kreider, R. B., Forbes, S. C., et al. (2025) · Journal of the International Society of Sports Nutrition · doi:10.1080/15502783.2025.2534130 · Cited by 43
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Volkhin, I. A., Paremskaia, A. I., Dashian, M. A., Smeshnova, D. S., Pavlov, R. E., Mityaeva, O. N., Volchkov, P. Y., & Deviatkin, A. A. (2025) · Biochemistry (Moscow) · doi:10.1134/s0006297924604659 · Cited by 7
Metadata
- Authors
- Strauss, K. A., Farrar, M. A., Muntoni, F., Saito, K., Mendell, J. R., Servais, L., McMillan, H. J., Finkel, R. S., Swoboda, K. J., Kwon, J. M., Zaidman, C. M., Chiriboga, C. A., Iannaccone, S. T., Krueger, J. M., Parsons, J. A., Shieh, P. B., Kavanagh, S., Tauscher-Wisniewski, S., McGill, B. E., & Macek, T. A.
- Venue
- Nature Medicine
- Published
- 2022-06-17
- Volume
- 28
- Issue
- 7
- Pages
- 1381-1389
- ISSN
- 1078-8956 (ISSN-L)
- 1546-170X
- OpenAlex
- W4283011798
- Cited by
- 316
- References
- 7
- FWCI
- 32.04
- Type
- journal article
- Language
- en
- Open access
- hybrid
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Bibliographic data: Semantic Scholar, OpenAlex, Crossref.
Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.
