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122 results

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Page 1
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: polydefkis m. 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.
Assessing mNIS+7Ionis and international neurologists' proficiency in a familial amyloidotic polyneuropathy trial.
Dyck PJ, Kincaid JC, Dyck PJB, Chaudhry V, Goyal NA, Alves C, Salhi H, Wiesman JF, Labeyrie C, Robinson-Papp J, Cardoso M, Laura M, Ruzhansky K, Cortese A, Brannagan TH 3rd, Khoury J, Khella S, Waddington-Cruz M, Ferreira J, Wang AK, Pinto MV, Ayache SS, Benson MD, Berk JL, Coelho T, Polydefkis M, Gorevic P, Adams DH, Plante-Bordeneuve V, Whelan C, Merlini G, Heitner S, Drachman BM, Conceição I, Klein CJ, Gertz MA, Ackermann EJ, Hughes SG, Mauermann ML, Bergemann R, Lodermeier KA, Davies JL, Carter RE, Litchy WJ. Dyck PJ, et al. Among authors: polydefkis m. Muscle Nerve. 2017 Nov;56(5):901-911. doi: 10.1002/mus.25563. Epub 2017 Apr 7. Muscle Nerve. 2017. PMID: 28063170 Free PMC article. Clinical Trial.
Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis.
Benson MD, Waddington-Cruz M, Berk JL, Polydefkis M, Dyck PJ, Wang AK, Planté-Bordeneuve V, Barroso FA, Merlini G, Obici L, Scheinberg M, Brannagan TH 3rd, Litchy WJ, Whelan C, Drachman BM, Adams D, Heitner SB, Conceição I, Schmidt HH, Vita G, Campistol JM, Gamez J, Gorevic PD, Gane E, Shah AM, Solomon SD, Monia BP, Hughes SG, Kwoh TJ, McEvoy BW, Jung SW, Baker BF, Ackermann EJ, Gertz MA, Coelho T. Benson MD, et al. Among authors: polydefkis m. N Engl J Med. 2018 Jul 5;379(1):22-31. doi: 10.1056/NEJMoa1716793. N Engl J Med. 2018. PMID: 29972757 Free article. Clinical Trial.
Hereditary transthyretin amyloidosis: baseline characteristics of patients in the NEURO-TTR trial.
Waddington-Cruz M, Ackermann EJ, Polydefkis M, Heitner SB, Dyck PJ, Barroso FA, Wang AK, Berk JL, Dyck PJB, Monia BP, Hughes SG, Tai L, Jesse Kwoh T, Jung SW, Coelho T, Benson MD, Gertz MA. Waddington-Cruz M, et al. Among authors: polydefkis m. Amyloid. 2018 Sep;25(3):180-188. doi: 10.1080/13506129.2018.1503593. Epub 2018 Aug 31. Amyloid. 2018. PMID: 30169969 Free article. Clinical Trial.
Oligonucleotide Drugs for Transthyretin Amyloidosis.
Adams D, Hawkins PN, Polydefkis M. Adams D, et al. Among authors: polydefkis m. N Engl J Med. 2018 Nov 22;379(21):2086. doi: 10.1056/NEJMc1810994. N Engl J Med. 2018. PMID: 30484624 No abstract available.
An indirect treatment comparison of the efficacy of patisiran and tafamidis for the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy.
Planté-Bordeneuve V, Lin H, Gollob J, Agarwal S, Betts M, Fahrbach K, Chitnis M, Polydefkis M. Planté-Bordeneuve V, et al. Among authors: polydefkis m. Expert Opin Pharmacother. 2019 Mar;20(4):473-481. doi: 10.1080/14656566.2018.1554648. Epub 2018 Dec 12. Expert Opin Pharmacother. 2019. PMID: 30489166 Review.
Response to the letter to the editor on "an indirect treatment comparison of the efficacy of patisiran and tafamidis for the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy".
Lin H, Agarwal S, Betts M, Fahrbach K, Chitnis M, Polydefkis M. Lin H, et al. Among authors: polydefkis m. Expert Opin Pharmacother. 2019 Aug;20(12):1529-1530. doi: 10.1080/14656566.2019.1620987. Epub 2019 May 24. Expert Opin Pharmacother. 2019. PMID: 31124741 No abstract available.
Analysis of autonomic outcomes in APOLLO, a phase III trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis.
González-Duarte A, Berk JL, Quan D, Mauermann ML, Schmidt HH, Polydefkis M, Waddington-Cruz M, Ueda M, Conceição IM, Kristen AV, Coelho T, Cauquil CA, Tard C, Merkel M, Aldinc E, Chen J, Sweetser MT, Wang JJ, Adams D. González-Duarte A, et al. Among authors: polydefkis m. J Neurol. 2020 Mar;267(3):703-712. doi: 10.1007/s00415-019-09602-8. Epub 2019 Nov 14. J Neurol. 2020. PMID: 31728713 Free PMC article. Clinical Trial.
122 results