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Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: 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, Wigderson M, Tauscher-Wisniewski S, McGill BE, Macek TA. Strauss KA, et al. Among authors: iannaccone st. Nat Med. 2022 Jul;28(7):1390-1397. doi: 10.1038/s41591-022-01867-3. Epub 2022 Jun 17. Nat Med. 2022. PMID: 35715567 Free PMC article. Clinical Trial.
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.
Rasch analysis of clinical outcome measures in spinal muscular atrophy.
Cano SJ, Mayhew A, Glanzman AM, Krosschell KJ, Swoboda KJ, Main M, Steffensen BF, Bérard C, Girardot F, Payan CA, Mercuri E, Mazzone E, Elsheikh B, Florence J, Hynan LS, Iannaccone ST, Nelson LL, Pandya S, Rose M, Scott C, Sadjadi R, Yore MA, Joyce C, Kissel JT; International Coordinating Committee for SMA Clinical Trials Rasch Task Force. Cano SJ, et al. Among authors: iannaccone st. Muscle Nerve. 2014 Mar;49(3):422-30. doi: 10.1002/mus.23937. Epub 2013 Jul 26. Muscle Nerve. 2014. PMID: 23836324 Free PMC article.
Diagnostic approach to the congenital muscular dystrophies.
Bönnemann CG, Wang CH, Quijano-Roy S, Deconinck N, Bertini E, Ferreiro A, Muntoni F, Sewry C, Béroud C, Mathews KD, Moore SA, Bellini J, Rutkowski A, North KN; Members of International Standard of Care Committee for Congenital Muscular Dystrophies. Bönnemann CG, et al. Neuromuscul Disord. 2014 Apr;24(4):289-311. doi: 10.1016/j.nmd.2013.12.011. Epub 2014 Jan 9. Neuromuscul Disord. 2014. PMID: 24581957 Free PMC article.
166 results