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Corrigendum to 'Population pharmacokinetics-based recommendations for a single delayed or missed dose of nusinersen': Neuromuscular Disorders 31 (2021) 310-318/doi: 10.1016/j.nmd.2021.02.014.
MacCannell D, Berger Z, East L, Mercuri E, Kirschner J, Muntoni F, Farrar MA, Peng J, Zhou J, Nestorov I, Farwell W, Finkel RS. MacCannell D, et al. Among authors: farrar ma. Neuromuscul Disord. 2022 Jan;32(1):e1. doi: 10.1016/j.nmd.2022.01.002. Neuromuscul Disord. 2022. PMID: 35144755 Free article. No abstract available.
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.
Finkel RS, Mercuri E, Darras BT, Connolly AM, Kuntz NL, Kirschner J, Chiriboga CA, Saito K, Servais L, Tizzano E, Topaloglu H, Tulinius M, Montes J, Glanzman AM, Bishop K, Zhong ZJ, Gheuens S, Bennett CF, Schneider E, Farwell W, De Vivo DC; ENDEAR Study Group. Finkel RS, et al. N Engl J Med. 2017 Nov 2;377(18):1723-1732. doi: 10.1056/NEJMoa1702752. N Engl J Med. 2017. PMID: 29091570 Free article. Clinical Trial.
Restoration of Nusinersen Levels Following Treatment Interruption in People With Spinal Muscular Atrophy: Simulations Based on a Population Pharmacokinetic Model.
MacCannell D, Berger Z, Kirschner J, Mercuri E, Farrar MA, Iannaccone ST, Kuntz NL, Finkel RS, Valente M, Muntoni F. MacCannell D, et al. Among authors: farrar ma. CNS Drugs. 2022 Feb;36(2):181-190. doi: 10.1007/s40263-022-00899-0. Epub 2022 Jan 26. CNS Drugs. 2022. PMID: 35080757 Free PMC article.
Clinical practice guideline for the management of paediatric Charcot-Marie-Tooth disease.
Yiu EM, Bray P, Baets J, Baker SK, Barisic N, de Valle K, Estilow T, Farrar MA, Finkel RS, Haberlová J, Kennedy RA, Moroni I, Nicholson GA, Ramchandren S, Reilly MM, Rose K, Shy ME, Siskind CE, Yum SW, Menezes MP, Ryan MM, Burns J. Yiu EM, et al. Among authors: farrar ma. J Neurol Neurosurg Psychiatry. 2022 May;93(5):530-538. doi: 10.1136/jnnp-2021-328483. Epub 2022 Feb 9. J Neurol Neurosurg Psychiatry. 2022. PMID: 35140138 Review.
Onasemnogene abeparvovec for the treatment of spinal muscular atrophy.
McMillan HJ, Proud CM, Farrar MA, Alexander IE, Muntoni F, Servais L. McMillan HJ, et al. Among authors: farrar ma. Expert Opin Biol Ther. 2022 Sep;22(9):1075-1090. doi: 10.1080/14712598.2022.2066471. Epub 2022 May 2. Expert Opin Biol Ther. 2022. PMID: 35437095 Free article.
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: farrar ma. 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.
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: farrar ma. 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.
230 results