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523 results

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Page 1
A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis.
Coelho T, Adams D, Conceição I, Waddington-Cruz M, Schmidt HH, Buades J, Campistol J, Berk JL, Polydefkis M, Wang JJ, Chen J, Sweetser MT, Gollob J, Suhr OB. Coelho T, et al. Among authors: schmidt hh. Orphanet J Rare Dis. 2020 Jul 8;15(1):179. doi: 10.1186/s13023-020-01399-4. Orphanet J Rare Dis. 2020. PMID: 32641071 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: schmidt hh. 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.
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: schmidt hh. 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.
Inotersen preserves or improves quality of life in hereditary transthyretin amyloidosis.
Coelho T, Yarlas A, Waddington-Cruz M, White MK, Sikora Kessler A, Lovley A, Pollock M, Guthrie S, Ackermann EJ, Hughes SG, Karam C, Khella S, Gertz M, Merlini G, Obici L, Schmidt HH, Polydefkis M, Dyck PJB, Brannagan Iii TH, Conceição I, Benson MD, Berk JL. Coelho T, et al. Among authors: schmidt hh. J Neurol. 2020 Apr;267(4):1070-1079. doi: 10.1007/s00415-019-09671-9. Epub 2019 Dec 18. J Neurol. 2020. PMID: 31853709 Free PMC article. Clinical Trial.
Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study.
Adams D, Polydefkis M, González-Duarte A, Wixner J, Kristen AV, Schmidt HH, Berk JL, Losada López IA, Dispenzieri A, Quan D, Conceição IM, Slama MS, Gillmore JD, Kyriakides T, Ajroud-Driss S, Waddington-Cruz M, Mezei MM, Planté-Bordeneuve V, Attarian S, Mauricio E, Brannagan TH 3rd, Ueda M, Aldinc E, Wang JJ, White MT, Vest J, Berber E, Sweetser MT, Coelho T; patisiran Global OLE study group. Adams D, et al. Among authors: schmidt hh. Lancet Neurol. 2021 Jan;20(1):49-59. doi: 10.1016/S1474-4422(20)30368-9. Epub 2020 Nov 16. Lancet Neurol. 2021. PMID: 33212063 Clinical Trial.
Quality of life outcomes in APOLLO, the phase 3 trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis.
Obici L, Berk JL, González-Duarte A, Coelho T, Gillmore J, Schmidt HH, Schilling M, Yamashita T, Labeyrie C, Brannagan TH 3rd, Ajroud-Driss S, Gorevic P, Kristen AV, Franklin J, Chen J, Sweetser MT, Wang JJ, Adams D. Obici L, et al. Among authors: schmidt hh. Amyloid. 2020 Sep;27(3):153-162. doi: 10.1080/13506129.2020.1730790. Epub 2020 Mar 4. Amyloid. 2020. PMID: 32131641 Clinical Trial.
Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial.
Coelho T, Maia LF, Martins da Silva A, Waddington Cruz M, Planté-Bordeneuve V, Lozeron P, Suhr OB, Campistol JM, Conceição IM, Schmidt HH, Trigo P, Kelly JW, Labaudinière R, Chan J, Packman J, Wilson A, Grogan DR. Coelho T, et al. Among authors: schmidt hh. Neurology. 2012 Aug 21;79(8):785-92. doi: 10.1212/WNL.0b013e3182661eb1. Epub 2012 Jul 25. Neurology. 2012. PMID: 22843282 Free PMC article. Clinical Trial.
Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy.
Coelho T, Maia LF, da Silva AM, Cruz MW, Planté-Bordeneuve V, Suhr OB, Conceiçao I, Schmidt HH, Trigo P, Kelly JW, Labaudinière R, Chan J, Packman J, Grogan DR. Coelho T, et al. Among authors: schmidt hh. J Neurol. 2013 Nov;260(11):2802-14. doi: 10.1007/s00415-013-7051-7. Epub 2013 Aug 22. J Neurol. 2013. PMID: 23974642 Free PMC article. Clinical Trial.
Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial.
Adams D, Tournev IL, Taylor MS, Coelho T, Planté-Bordeneuve V, Berk JL, González-Duarte A, Gillmore JD, Low SC, Sekijima Y, Obici L, Chen C, Badri P, Arum SM, Vest J, Polydefkis M; HELIOS-A Collaborators. Adams D, et al. Amyloid. 2023 Mar;30(1):1-9. doi: 10.1080/13506129.2022.2091985. Epub 2022 Jul 23. Amyloid. 2023. PMID: 35875890 Clinical Trial.
Correction to: 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: schmidt hh. J Neurol. 2020 Mar;267(3):713-714. doi: 10.1007/s00415-020-09715-5. J Neurol. 2020. PMID: 32030522 Free PMC article.
523 results