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2,626 results

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Page 1
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. Among authors: schneider e. 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.
Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy.
Mercuri E, Darras BT, Chiriboga CA, Day JW, Campbell C, Connolly AM, Iannaccone ST, Kirschner J, Kuntz NL, Saito K, Shieh PB, Tulinius M, Mazzone ES, Montes J, Bishop KM, Yang Q, Foster R, Gheuens S, Bennett CF, Farwell W, Schneider E, De Vivo DC, Finkel RS; CHERISH Study Group. Mercuri E, et al. Among authors: schneider e. N Engl J Med. 2018 Feb 15;378(7):625-635. doi: 10.1056/NEJMoa1710504. N Engl J Med. 2018. PMID: 29443664 Free article. Clinical Trial.
Nusinersen in later-onset spinal muscular atrophy: Long-term results from the phase 1/2 studies.
Darras BT, Chiriboga CA, Iannaccone ST, Swoboda KJ, Montes J, Mignon L, Xia S, Bennett CF, Bishop KM, Shefner JM, Green AM, Sun P, Bhan I, Gheuens S, Schneider E, Farwell W, De Vivo DC; ISIS-396443-CS2/ISIS-396443-CS12 Study Groups. Darras BT, et al. Among authors: schneider e. Neurology. 2019 May 21;92(21):e2492-e2506. doi: 10.1212/WNL.0000000000007527. Epub 2019 Apr 24. Neurology. 2019. PMID: 31019106 Free PMC article. Clinical Trial.
Nusinersen improves walking distance and reduces fatigue in later-onset spinal muscular atrophy.
Montes J, Dunaway Young S, Mazzone ES, Pasternak A, Glanzman AM, Finkel RS, Darras BT, Muntoni F, Mercuri E, De Vivo DC, Bishop KM, Schneider E, Bennett CF, Foster R, Farwell W; CS2 and CS12 Study Groups. Montes J, et al. Among authors: schneider e. Muscle Nerve. 2019 Oct;60(4):409-414. doi: 10.1002/mus.26633. Epub 2019 Jul 27. Muscle Nerve. 2019. PMID: 31298747 Free PMC article. Clinical Trial.
Antisense Inhibition of Prekallikrein to Control Hereditary Angioedema.
Cohn DM, Viney NJ, Fijen LM, Schneider E, Alexander VJ, Xia S, Kaeser GE, Nanavati C, Baker BF, Geary RS, Levi M, Meijers JCM, Stroes ESG. Cohn DM, et al. Among authors: schneider e. N Engl J Med. 2020 Sep 24;383(13):1242-1247. doi: 10.1056/NEJMoa1915035. Epub 2020 Sep 2. N Engl J Med. 2020. PMID: 32877578 Clinical Trial.
Treatment of infantile-onset spinal muscular atrophy with nusinersen: final report of a phase 2, open-label, multicentre, dose-escalation study.
Finkel RS, Chiriboga CA, Vajsar J, Day JW, Montes J, De Vivo DC, Bishop KM, Foster R, Liu Y, Ramirez-Schrempp D, Schneider E, Bennett CF, Wong J, Farwell W. Finkel RS, et al. Among authors: schneider e. Lancet Child Adolesc Health. 2021 Jul;5(7):491-500. doi: 10.1016/S2352-4642(21)00100-0. Epub 2021 Jun 3. Lancet Child Adolesc Health. 2021. PMID: 34089650 Clinical Trial.
Inhibition of Prekallikrein for Hereditary Angioedema.
Fijen LM, Riedl MA, Bordone L, Bernstein JA, Raasch J, Tachdjian R, Craig T, Lumry WR, Manning ME, Alexander VJ, Newman KB, Revenko A, Baker BF, Nanavati C, MacLeod AR, Schneider E, Cohn DM. Fijen LM, et al. Among authors: schneider e. N Engl J Med. 2022 Mar 17;386(11):1026-1033. doi: 10.1056/NEJMoa2109329. N Engl J Med. 2022. PMID: 35294812 Clinical Trial.
Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data.
Viney NJ, Guo S, Tai LJ, Baker BF, Aghajan M, Jung SW, Yu RZ, Booten S, Murray H, Machemer T, Burel S, Murray S, Buchele G, Tsimikas S, Schneider E, Geary RS, Benson MD, Monia BP. Viney NJ, et al. Among authors: schneider e. ESC Heart Fail. 2021 Feb;8(1):652-661. doi: 10.1002/ehf2.13154. Epub 2020 Dec 7. ESC Heart Fail. 2021. PMID: 33283485 Free PMC article. Clinical Trial.
IONIS-PKKRx a Novel Antisense Inhibitor of Prekallikrein and Bradykinin Production.
Ferrone JD, Bhattacharjee G, Revenko AS, Zanardi TA, Warren MS, Derosier FJ, Viney NJ, Pham NC, Kaeser GE, Baker BF, Schneider E, Hughes SG, Monia BP, MacLeod AR. Ferrone JD, et al. Among authors: schneider e. Nucleic Acid Ther. 2019 Apr;29(2):82-91. doi: 10.1089/nat.2018.0754. Epub 2019 Feb 28. Nucleic Acid Ther. 2019. PMID: 30817230 Free PMC article.
2,626 results