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Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial.
Strauss KA, Farrar MA, Muntoni F, Saito K, Mendell JR, Servais L, McMillan HJ, Finkel RS, Swoboda KJ, Kwon JM, Zaidman CM, Chiriboga CA, Iannaccone ST, Krueger JM, Parsons JA, Shieh PB, Kavanagh S, Tauscher-Wisniewski S, McGill BE, Macek TA. Strauss KA, et al. Among authors: iannaccone st. Nat Med. 2022 Jul;28(7):1381-1389. doi: 10.1038/s41591-022-01866-4. Epub 2022 Jun 17. Nat Med. 2022. PMID: 35715566 Free PMC article. Clinical Trial.
Outcome measures for pediatric spinal muscular atrophy.
Iannaccone ST; American Spinal Muscular Atrophy Randomized Trials (AmSMART) Group. Iannaccone ST, et al. Arch Neurol. 2002 Sep;59(9):1445-50. doi: 10.1001/archneur.59.9.1445. Arch Neurol. 2002. PMID: 12223032
Spinal muscular atrophy.
Iannaccone ST, Smith SA, Simard LR. Iannaccone ST, et al. Curr Neurol Neurosci Rep. 2004 Jan;4(1):74-80. doi: 10.1007/s11910-004-0016-6. Curr Neurol Neurosci Rep. 2004. PMID: 14683633 Review.
Clinical trials in spinal muscular atrophy.
Kaufmann P, Iannaccone ST. Kaufmann P, et al. Among authors: iannaccone st. Phys Med Rehabil Clin N Am. 2008 Aug;19(3):653-60, xii. doi: 10.1016/j.pmr.2008.04.006. Phys Med Rehabil Clin N Am. 2008. PMID: 18625422 Review.
166 results