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Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis.
Adams D, Gonzalez-Duarte A, O'Riordan WD, Yang CC, Ueda M, Kristen AV, Tournev I, Schmidt HH, Coelho T, Berk JL, Lin KP, Vita G, Attarian S, Planté-Bordeneuve V, Mezei MM, Campistol JM, Buades J, Brannagan TH 3rd, Kim BJ, Oh J, Parman Y, Sekijima Y, Hawkins PN, Solomon SD, Polydefkis M, Dyck PJ, Gandhi PJ, Goyal S, Chen J, Strahs AL, Nochur SV, Sweetser MT, Garg PP, Vaishnaw AK, Gollob JA, Suhr OB. Adams D, et al. Among authors: goyal s. N Engl J Med. 2018 Jul 5;379(1):11-21. doi: 10.1056/NEJMoa1716153. N Engl J Med. 2018. PMID: 29972753 Free article. Clinical Trial.
Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease.
Kanter J, Walters MC, Krishnamurti L, Mapara MY, Kwiatkowski JL, Rifkin-Zenenberg S, Aygun B, Kasow KA, Pierciey FJ Jr, Bonner M, Miller A, Zhang X, Lynch J, Kim D, Ribeil JA, Asmal M, Goyal S, Thompson AA, Tisdale JF. Kanter J, et al. Among authors: goyal s. N Engl J Med. 2022 Feb 17;386(7):617-628. doi: 10.1056/NEJMoa2117175. Epub 2021 Dec 12. N Engl J Med. 2022. PMID: 34898139 Clinical Trial.
Acute Myeloid Leukemia Case after Gene Therapy for Sickle Cell Disease.
Goyal S, Tisdale J, Schmidt M, Kanter J, Jaroscak J, Whitney D, Bitter H, Gregory PD, Parsons G, Foos M, Yeri A, Gioia M, Voytek SB, Miller A, Lynch J, Colvin RA, Bonner M. Goyal S, et al. N Engl J Med. 2022 Jan 13;386(2):138-147. doi: 10.1056/NEJMoa2109167. Epub 2021 Dec 12. N Engl J Med. 2022. PMID: 34898140
2,144 results